Jingjing Liu, Zhifa Wang, Nan Wang, Jingyao Ma, Yu Hu, Jie Ma, Lijuan Wang, Yan Liu, Juntao Ouyang, Zhenping Chen, Xiaoling Cheng, Runhui Wu
<p>Immune thrombocytopenia (ITP) is an acquired bleeding disorder characterized by a reduced platelet count of less than 100 × 10<sup>9</sup>/L, with an estimated incidence of 2–5/100 000 children per year [<span>1</span>]. Since 2014, the China National Medical Products Administration (NMPA) has approved five TPO-RAs for treating ITP in China. Among these, eltrombopag (ELT) and avatrombopag (AVA) are the two most evidence-based and available oral medications for pediatric ITP; both demonstrated promising efficacy and tolerability in clinical trials [<span>2, 3</span>]. Because no head-to-head studies have been conducted, making direct comparisons between the two drugs is difficult. To address this gap, we conducted a multicenter, retrospective, observational cohort study to evaluate the efficacy and safety of ELT versus AVA in pediatric patients with ITP in China, to offer personalized treatment guidance based on factors such as the severity of bleeding, medication adherence, and family economic conditions.</p> <p>Patients younger than 18 with a confirmed diagnosis of ITP who received treatment with ELT or AVA in 3 Children's Specialty Hospitals from April 2017 to December 2023, with medication duration for at least 2 months and with a total observation period of more than 6 months. For this retrospective study, ethics approval was obtained from the institutional review boards of Beijing Children's Hospital, Capital Medical University (Ethics approval NO. [2024]-Y-098-D).</p> <p>The initial dosage of ELT was determined by age and body weight. For children aged 1–5 years, the starting dose was 1.5 mg/kg/day. In those aged 6–17 years, the dose was 37.5 mg/day for individuals weighing less than 27 kg and 50 mg/day for those weighing 27 kg or more. Similarly, the starting dose of AVA was 10 mg/day for children aged 1–6 years and 20 mg/day for those aged 6–18 years.</p> <p>The current study defines several key definitions and outcomes [<span>4</span>]:(1) Complete Response (CR): a platelet count ≥ 100 × 10<sup>9</sup>/L and no bleeding within 2 months. (2) Response (R): a platelet count of 30–100 × 10<sup>9</sup>/L, with at least a twofold increase from baseline and no bleeding within 2 months. (3) No Response (NR): a platelet count < 30 × 10<sup>9</sup>/L, less than a twofold increase from baseline, or bleeding after dose titration of ELT or AVA within 2 months. (4) Overall Response (OR): the combined number of patients achieving CR and R. (5) Durable Response (DR): a platelet count > 30 × 10<sup>9</sup>/L with at least a twofold increase from baseline at 6 months. (6) Remission: a platelet count ≥ 30 × 10<sup>9</sup>/L at 12 months. (7) SRoT: a platelet count ≥ 30 × 10<sup>9</sup>/L for at least 6 months off ELT or AVA treatment. (8) Time to typer: defined as the period from the initiation of ELT/AVA therapy to the commencement of dosage tapering within the first 12 months. (9) Rescue therapy, including IVIG, platelet transfusion, and high-do
{"title":"Clinical Use of Eltrombopag and Avatrombopag in Pediatric ITP in China: A Real-World Multicenter Retrospective Cohort Study","authors":"Jingjing Liu, Zhifa Wang, Nan Wang, Jingyao Ma, Yu Hu, Jie Ma, Lijuan Wang, Yan Liu, Juntao Ouyang, Zhenping Chen, Xiaoling Cheng, Runhui Wu","doi":"10.1002/ajh.27554","DOIUrl":"https://doi.org/10.1002/ajh.27554","url":null,"abstract":"<p>Immune thrombocytopenia (ITP) is an acquired bleeding disorder characterized by a reduced platelet count of less than 100 × 10<sup>9</sup>/L, with an estimated incidence of 2–5/100 000 children per year [<span>1</span>]. Since 2014, the China National Medical Products Administration (NMPA) has approved five TPO-RAs for treating ITP in China. Among these, eltrombopag (ELT) and avatrombopag (AVA) are the two most evidence-based and available oral medications for pediatric ITP; both demonstrated promising efficacy and tolerability in clinical trials [<span>2, 3</span>]. Because no head-to-head studies have been conducted, making direct comparisons between the two drugs is difficult. To address this gap, we conducted a multicenter, retrospective, observational cohort study to evaluate the efficacy and safety of ELT versus AVA in pediatric patients with ITP in China, to offer personalized treatment guidance based on factors such as the severity of bleeding, medication adherence, and family economic conditions.</p>\u0000<p>Patients younger than 18 with a confirmed diagnosis of ITP who received treatment with ELT or AVA in 3 Children's Specialty Hospitals from April 2017 to December 2023, with medication duration for at least 2 months and with a total observation period of more than 6 months. For this retrospective study, ethics approval was obtained from the institutional review boards of Beijing Children's Hospital, Capital Medical University (Ethics approval NO. [2024]-Y-098-D).</p>\u0000<p>The initial dosage of ELT was determined by age and body weight. For children aged 1–5 years, the starting dose was 1.5 mg/kg/day. In those aged 6–17 years, the dose was 37.5 mg/day for individuals weighing less than 27 kg and 50 mg/day for those weighing 27 kg or more. Similarly, the starting dose of AVA was 10 mg/day for children aged 1–6 years and 20 mg/day for those aged 6–18 years.</p>\u0000<p>The current study defines several key definitions and outcomes [<span>4</span>]:(1) Complete Response (CR): a platelet count ≥ 100 × 10<sup>9</sup>/L and no bleeding within 2 months. (2) Response (R): a platelet count of 30–100 × 10<sup>9</sup>/L, with at least a twofold increase from baseline and no bleeding within 2 months. (3) No Response (NR): a platelet count < 30 × 10<sup>9</sup>/L, less than a twofold increase from baseline, or bleeding after dose titration of ELT or AVA within 2 months. (4) Overall Response (OR): the combined number of patients achieving CR and R. (5) Durable Response (DR): a platelet count > 30 × 10<sup>9</sup>/L with at least a twofold increase from baseline at 6 months. (6) Remission: a platelet count ≥ 30 × 10<sup>9</sup>/L at 12 months. (7) SRoT: a platelet count ≥ 30 × 10<sup>9</sup>/L for at least 6 months off ELT or AVA treatment. (8) Time to typer: defined as the period from the initiation of ELT/AVA therapy to the commencement of dosage tapering within the first 12 months. (9) Rescue therapy, including IVIG, platelet transfusion, and high-do","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"48 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142841749","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Romain Ravel-Chapuis, Gérard Buchonnet, Catherine Boutet, Francine Garnache Ottou, Anne Roggy, Florian Renosi, Olivier Matray, Victor Bobée
<p>A 60-year-old male presented with spontaneous hematomas and cervical adenopathy. Laboratory workup revealed hemoglobin 12.3 g/dL, platelet 56 × 10<sup>9</sup>/L, and leukocyte 14.7 × 10<sup>9</sup>/L. The automated cell counter (Sysmex XN) reported 9% neutrophils (1.32 × 10<sup>9</sup>/L) and 86% lymphocytes (12.6 × 10<sup>9</sup>/L), suggestive of a lymphoproliferative disorder. Blood smear showed atypical lymphoid cells with normal size, mature chromatin, and scant cytoplasm Figure 1 (Panel A), while numerous lysed cells were also observed (Panel B). About 10% of these cells displayed cytoplasmic vacuoles, occasionally coalescing, and 10% appeared larger with grayish cytoplasm (Panel C and D). Due to the apparent lymphocytosis, flow cytometry immunophenotyping was performed (Navios EX, Beckman-Coulter). Surprisingly, 62% of the leukocytes consisted of a CD45+low blastic population. These cells were negative for common lymphoid (CD19/CD20/CD22/CD24/cCD79a/CD3/CD5/CD8/CD10) and myeloid antigens (CD34/CD38/CD13/CD14/CD42b/CD61/CD64/CD65/CD117/cMPO), but were positive for CD4(weak)/CD56/CD123(strong)/HLA-DR(strong)/CD33 (Panel E and F). A cutaneous lesion was found on the patient's back, and bone marrow aspiration confirmed 82% of the same population with additional phenotypic markers indicative of pDC lineage (CD303+weak/CD304+/cTCL1+/BadLamp+), supporting the diagnosis of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). The neurological examination was normal, and cerebrospinal involvement was inconclusive due to hemorrhagic cerebrospinal fluid samples. Furthermore, the mutational profile performed on bone marrow aspirate was consistent with BPDCN, revealing mutations in <i>TET2, ASXL1</i>, and <i>NF1</i>, as well as a subclonal <i>NRAS</i> mutation and deletions in the <i>IKZF1</i> and <i>ETV6</i> loci.</p> <p>BPDCN is a rare hematologic malignancy, with diverse morphological presentations. While the classic blastic morphology, often exhibiting “pearl necklace” cytoplasmic appearances and “hand-mirror-like” projections, is the most common presentation, this case displayed an unusual morphology dominated by pseudo-lymphoid cells. Although a subset of pseudo-lymphoid cells is commonly observed in BPDCN, this case was remarkable in that nearly all cells exhibited this distinctive morphology. Such atypical presentation can be deceptive, initially mimicking lymphoid malignancies. Here, the immunophenotypic profile was crucial in establishing the accurate diagnosis.</p> <figure><picture> <source media="(min-width: 1650px)" srcset="/cms/asset/066ddd2e-9d43-407c-8ef9-7a0c7829025e/ajh27567-fig-0001-m.jpg"/><img alt="Details are in the caption following the image" data-lg-src="/cms/asset/066ddd2e-9d43-407c-8ef9-7a0c7829025e/ajh27567-fig-0001-m.jpg" loading="lazy" src="/cms/asset/606f65ba-04c1-4c37-bab4-d263c99fa856/ajh27567-fig-0001-m.png" title="Details are in the caption following the image"/></picture><figcaption> <div><strong>FIGURE 1<span sty
{"title":"Blastic Plasmocytoid Dendritic Cell Neoplasm With Pseudo-Lymphoid Morphology Mimicking Lymphoid Malignancy","authors":"Romain Ravel-Chapuis, Gérard Buchonnet, Catherine Boutet, Francine Garnache Ottou, Anne Roggy, Florian Renosi, Olivier Matray, Victor Bobée","doi":"10.1002/ajh.27567","DOIUrl":"https://doi.org/10.1002/ajh.27567","url":null,"abstract":"<p>A 60-year-old male presented with spontaneous hematomas and cervical adenopathy. Laboratory workup revealed hemoglobin 12.3 g/dL, platelet 56 × 10<sup>9</sup>/L, and leukocyte 14.7 × 10<sup>9</sup>/L. The automated cell counter (Sysmex XN) reported 9% neutrophils (1.32 × 10<sup>9</sup>/L) and 86% lymphocytes (12.6 × 10<sup>9</sup>/L), suggestive of a lymphoproliferative disorder. Blood smear showed atypical lymphoid cells with normal size, mature chromatin, and scant cytoplasm Figure 1 (Panel A), while numerous lysed cells were also observed (Panel B). About 10% of these cells displayed cytoplasmic vacuoles, occasionally coalescing, and 10% appeared larger with grayish cytoplasm (Panel C and D). Due to the apparent lymphocytosis, flow cytometry immunophenotyping was performed (Navios EX, Beckman-Coulter). Surprisingly, 62% of the leukocytes consisted of a CD45+low blastic population. These cells were negative for common lymphoid (CD19/CD20/CD22/CD24/cCD79a/CD3/CD5/CD8/CD10) and myeloid antigens (CD34/CD38/CD13/CD14/CD42b/CD61/CD64/CD65/CD117/cMPO), but were positive for CD4(weak)/CD56/CD123(strong)/HLA-DR(strong)/CD33 (Panel E and F). A cutaneous lesion was found on the patient's back, and bone marrow aspiration confirmed 82% of the same population with additional phenotypic markers indicative of pDC lineage (CD303+weak/CD304+/cTCL1+/BadLamp+), supporting the diagnosis of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). The neurological examination was normal, and cerebrospinal involvement was inconclusive due to hemorrhagic cerebrospinal fluid samples. Furthermore, the mutational profile performed on bone marrow aspirate was consistent with BPDCN, revealing mutations in <i>TET2, ASXL1</i>, and <i>NF1</i>, as well as a subclonal <i>NRAS</i> mutation and deletions in the <i>IKZF1</i> and <i>ETV6</i> loci.</p>\u0000<p>BPDCN is a rare hematologic malignancy, with diverse morphological presentations. While the classic blastic morphology, often exhibiting “pearl necklace” cytoplasmic appearances and “hand-mirror-like” projections, is the most common presentation, this case displayed an unusual morphology dominated by pseudo-lymphoid cells. Although a subset of pseudo-lymphoid cells is commonly observed in BPDCN, this case was remarkable in that nearly all cells exhibited this distinctive morphology. Such atypical presentation can be deceptive, initially mimicking lymphoid malignancies. Here, the immunophenotypic profile was crucial in establishing the accurate diagnosis.</p>\u0000<figure><picture>\u0000<source media=\"(min-width: 1650px)\" srcset=\"/cms/asset/066ddd2e-9d43-407c-8ef9-7a0c7829025e/ajh27567-fig-0001-m.jpg\"/><img alt=\"Details are in the caption following the image\" data-lg-src=\"/cms/asset/066ddd2e-9d43-407c-8ef9-7a0c7829025e/ajh27567-fig-0001-m.jpg\" loading=\"lazy\" src=\"/cms/asset/606f65ba-04c1-4c37-bab4-d263c99fa856/ajh27567-fig-0001-m.png\" title=\"Details are in the caption following the image\"/></picture><figcaption>\u0000<div><strong>FIGURE 1<span sty","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"1 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142841774","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Iliana Stamatiou, Zoe Bezirgiannidou, Evangelia Charitaki, Ioannis Kotsianidis, Konstantinos Liapis
<p>A 75-year-old woman presented to the emergency department with progressive abdominal pain, fever, and diarrhea after taking levofloxacin for a respiratory tract infection. On evaluation, she was in clinical shock, with blood pressure 88/58 mmHg and heart rate 122 beats per minute. A complete blood count provided by the Sysmex XN-1000 analyzer showed leukocytosis (13.8 × 10<sup>9</sup>/L, 95% neutrophils) and thrombocytopenia (22 × 10<sup>9</sup>/L). She had acidosis, renal impairment, coagulopathy, and elevated C-reactive protein level. Because of the thrombocytopenia, an examination of a peripheral-blood smear was performed in the hematology laboratory, which showed vacuolated neutrophils that contained phagocytized platelets. Of 200 neutrophils examined, 161 (80%) contained between one and six platelets (Figure 1). These findings indicated spurious thrombocytopenia. The emergency department staff were notified by the laboratory that the patient's platelet count was normal. Subsequently, she underwent internal jugular-vein catheterization for fluid resuscitation without oozing or hematoma. Pseudomembranous colitis was diagnosed on the basis of a positive <i>Clostridioides difficile</i> stool test. She was treated with metronidazole and vancomycin, but her course was complicated by renal failure necessitating hemodialysis. Eventually, she made a full recovery. During hospitalization, multiple routinely prepared films from EDTA-anticoagulated blood consistently demonstrated platelet phagocytosis but with the resolution of the colitis, the phenomenon became progressively less pronounced. The automated platelet count became normal within 30 days.</p> <figure><picture> <source media="(min-width: 1650px)" srcset="/cms/asset/43ba7b26-cdb9-4a52-851b-76e271182229/ajh27562-fig-0001-m.jpg"/><img alt="Details are in the caption following the image" data-lg-src="/cms/asset/43ba7b26-cdb9-4a52-851b-76e271182229/ajh27562-fig-0001-m.jpg" loading="lazy" src="/cms/asset/533e4fe2-c86d-4138-a00c-28ff5b938961/ajh27562-fig-0001-m.png" title="Details are in the caption following the image"/></picture><figcaption> <div><strong>FIGURE 1<span style="font-weight:normal"></span></strong><div>Open in figure viewer<i aria-hidden="true"></i><span>PowerPoint</span></div> </div> <div>Four fields of the peripheral-blood smear, showing ingestion of platelets by neutrophilic granulocytes (May-Grünwald-Giemsa stain, ×1000).</div> </figcaption> </figure> <p>A peripheral-blood smear should always be examined in new cases of thrombocytopenia or whenever the platelet count is unexpectedly low, in order to confirm the thrombocytopenia. The blood smear may be requested by physicians or initiated by laboratory staff [<span>1</span>]. As seen here, a laboratory-initiated blood smear, is particularly valuable because it may permit earlier recognition of pseudothrombocytopenia. The incidence of pseudothrombocytopenia is 1.9% among hospitalized patients and 0.15% in the outpatient setting [
{"title":"Pseudothrombocytopenia due to Phagocytosis of Platelets by Polymorphonuclear Leukocytes","authors":"Iliana Stamatiou, Zoe Bezirgiannidou, Evangelia Charitaki, Ioannis Kotsianidis, Konstantinos Liapis","doi":"10.1002/ajh.27562","DOIUrl":"https://doi.org/10.1002/ajh.27562","url":null,"abstract":"<p>A 75-year-old woman presented to the emergency department with progressive abdominal pain, fever, and diarrhea after taking levofloxacin for a respiratory tract infection. On evaluation, she was in clinical shock, with blood pressure 88/58 mmHg and heart rate 122 beats per minute. A complete blood count provided by the Sysmex XN-1000 analyzer showed leukocytosis (13.8 × 10<sup>9</sup>/L, 95% neutrophils) and thrombocytopenia (22 × 10<sup>9</sup>/L). She had acidosis, renal impairment, coagulopathy, and elevated C-reactive protein level. Because of the thrombocytopenia, an examination of a peripheral-blood smear was performed in the hematology laboratory, which showed vacuolated neutrophils that contained phagocytized platelets. Of 200 neutrophils examined, 161 (80%) contained between one and six platelets (Figure 1). These findings indicated spurious thrombocytopenia. The emergency department staff were notified by the laboratory that the patient's platelet count was normal. Subsequently, she underwent internal jugular-vein catheterization for fluid resuscitation without oozing or hematoma. Pseudomembranous colitis was diagnosed on the basis of a positive <i>Clostridioides difficile</i> stool test. She was treated with metronidazole and vancomycin, but her course was complicated by renal failure necessitating hemodialysis. Eventually, she made a full recovery. During hospitalization, multiple routinely prepared films from EDTA-anticoagulated blood consistently demonstrated platelet phagocytosis but with the resolution of the colitis, the phenomenon became progressively less pronounced. The automated platelet count became normal within 30 days.</p>\u0000<figure><picture>\u0000<source media=\"(min-width: 1650px)\" srcset=\"/cms/asset/43ba7b26-cdb9-4a52-851b-76e271182229/ajh27562-fig-0001-m.jpg\"/><img alt=\"Details are in the caption following the image\" data-lg-src=\"/cms/asset/43ba7b26-cdb9-4a52-851b-76e271182229/ajh27562-fig-0001-m.jpg\" loading=\"lazy\" src=\"/cms/asset/533e4fe2-c86d-4138-a00c-28ff5b938961/ajh27562-fig-0001-m.png\" title=\"Details are in the caption following the image\"/></picture><figcaption>\u0000<div><strong>FIGURE 1<span style=\"font-weight:normal\"></span></strong><div>Open in figure viewer<i aria-hidden=\"true\"></i><span>PowerPoint</span></div>\u0000</div>\u0000<div>Four fields of the peripheral-blood smear, showing ingestion of platelets by neutrophilic granulocytes (May-Grünwald-Giemsa stain, ×1000).</div>\u0000</figcaption>\u0000</figure>\u0000<p>A peripheral-blood smear should always be examined in new cases of thrombocytopenia or whenever the platelet count is unexpectedly low, in order to confirm the thrombocytopenia. The blood smear may be requested by physicians or initiated by laboratory staff [<span>1</span>]. As seen here, a laboratory-initiated blood smear, is particularly valuable because it may permit earlier recognition of pseudothrombocytopenia. The incidence of pseudothrombocytopenia is 1.9% among hospitalized patients and 0.15% in the outpatient setting [","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"26 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142825503","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Spiculated erythrocytes come in a variety of forms, which are of diagnostic importance. They can be broadly classified as echinocytes, acanthocytes, keratocytes, and schistocytes. However, complexity is added by the superimposition of one abnormality on another. Other spiculated cells can, for example, undergo an echinocytic or acanthocytic change. In addition, other poikilocytes, for example, spherocytes, can also undergo spiculation.
An acanthocyte is an erythrocyte with a small number of irregularly shaped and irregularly disposed projections on its surface. The name comes from the Greek, άκανθα, meaning thorn. These cells are most often a feature of hyposplenism but also occur in liver failure, when the term “spur cell hemolytic anemia” has been used. The upper images show acanthocytosis in a patient with drug-induced acute liver failure (all images May–Grunwald–Giemsa, ×100 objective). Acanthocytes also occur in rare inherited disorders such as the McLeod phenotype and choreo-acanthocytosis [1]. The lower images show a more complex situation. Here we have spheroacanthocytes in a patient with hereditary spherocytosis who has had a splenectomy. The formation of the spicules has been constrained by the spherical form of the cell.
An echinocyte is a cell with numerous short regular spicules. The name comes from the Greek, εχĩνος meaning sea-urchin. Echinocytosis is most often seen as a storage artifact, when it is referred to as crenation. It is a feature of pyruvate kinase deficiency, particularly post-splenectomy when the defective cells persist in the circulation [2].
A keratocyte is a cell with two or four paired spicules, from the Greek, κέρας, for horn, while a schistocyte is a jagged red cell fragment, from the Greek, σχίζω, for split or divide. Both are features of microangiopathic and mechanical hemolytic anemias [3]. Keratocytes are also seen in Heinz body hemolytic anemia, as in acute hemolysis in glucose-6-phosphate dehydrogenase deficiency.
An appreciation of these diverse spiculated cells can be important in diagnosis.
{"title":"The Diversity of Spiculated Erythrocytes","authors":"Barbara J. Bain","doi":"10.1002/ajh.27560","DOIUrl":"https://doi.org/10.1002/ajh.27560","url":null,"abstract":"<p><img alt=\"image\" loading=\"lazy\" src=\"/cms/asset/2cd87167-ea76-439c-a36d-dcebdb4994e9/ajh27560-gra-0001.png\"/>Spiculated erythrocytes come in a variety of forms, which are of diagnostic importance. They can be broadly classified as echinocytes, acanthocytes, keratocytes, and schistocytes. However, complexity is added by the superimposition of one abnormality on another. Other spiculated cells can, for example, undergo an echinocytic or acanthocytic change. In addition, other poikilocytes, for example, spherocytes, can also undergo spiculation.</p>\u0000<p>An acanthocyte is an erythrocyte with a small number of irregularly shaped and irregularly disposed projections on its surface. The name comes from the Greek, <i>άκανθα</i>, meaning thorn. These cells are most often a feature of hyposplenism but also occur in liver failure, when the term “spur cell hemolytic anemia” has been used. The upper images show acanthocytosis in a patient with drug-induced acute liver failure (all images May–Grunwald–Giemsa, ×100 objective). Acanthocytes also occur in rare inherited disorders such as the McLeod phenotype and choreo-acanthocytosis [<span>1</span>]. The lower images show a more complex situation. Here we have spheroacanthocytes in a patient with hereditary spherocytosis who has had a splenectomy. The formation of the spicules has been constrained by the spherical form of the cell.</p>\u0000<p>An echinocyte is a cell with numerous short regular spicules. The name comes from the Greek, <i>εχĩνος</i> meaning sea-urchin. Echinocytosis is most often seen as a storage artifact, when it is referred to as crenation. It is a feature of pyruvate kinase deficiency, particularly post-splenectomy when the defective cells persist in the circulation [<span>2</span>].</p>\u0000<p>A keratocyte is a cell with two or four paired spicules, from the Greek, <i>κέρα</i>ς, for horn, while a schistocyte is a jagged red cell fragment, from the Greek, <i>σχίζω</i>, for split or divide. Both are features of microangiopathic and mechanical hemolytic anemias [<span>3</span>]. Keratocytes are also seen in Heinz body hemolytic anemia, as in acute hemolysis in glucose-6-phosphate dehydrogenase deficiency.</p>\u0000<p>An appreciation of these diverse spiculated cells can be important in diagnosis.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"22 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142816211","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Naseema Gangat, Azeem Elbeih, Nour Ghosoun, Kristen McCullough, Fnu Aperna, Isla M. Johnson, Maymona Abdelmagid, Aref Al-Kali, Hassan B. Alkhateeb, Kebede H. Begna, Michelle Elliott, Abhishek Mangaonkar, Aasiya Matin, Antoine N. Saliba, Mehrdad Hefazi Torghabeh, Mark R. Litzow, William Hogan, Mithun Shah, Mrinal M. Patnaik, Animesh Pardanani, Talha Badar, Hemant Murthy, James Foran, Jeanne Palmer, Lisa Sproat, Nandita Khera, Cecilia Arana Yi, Samuel Yates, Abigail Sneider, Emily Dworkin, Anand A. Patel, Alexandre Bazinet, Jayastu Senapati, Alex Bataller, Courtney DiNardo, Tapan Kadia, Ayalew Tefferi
Patients with newly diagnosed acute myeloid leukemia (ND-AML) derive variable survival benefit from venetoclax + hypomethylating agent (Ven-HMA) therapy. The primary objective in the current study was to develop genetic risk models that are predictive of survival and are applicable at the time of diagnosis and after establishing treatment response. Among 400 ND-AML patients treated with Ven-HMA at the Mayo Clinic, 247 (62%) achieved complete remission with (CR) or without (CRi) count recovery. Multivariable analysis–derived hazard ratios (HR), including 1.8 for European LeukemiaNet (ELN) adverse karyotype, 4.7 for KMT2Ar, 1.7 for TP53MUT, 2.6 for KRASMUT, and 2.1 for IDH2WT were applied to develop an HR-weighted risk model: low, intermediate, and high; respective median survival censored for allogeneic stem cell transplant (ASCT) (3-year survival) were “not reached” (67%), 19.1 (33%), and 7.1 months (0%). In patients achieving CR/CRi, adverse karyotype, KMT2Ar, KRASMUT, IDH2WT predicted inferior survival, allowing for a complementary response-stratified risk model. The model was externally validated and was shown to be superior to the ELN 2024 risk model (AIC 179 vs. 195 and AUC 0.77 vs. 0.69). Survival was inferior with failure to achieve CR/CRi or not receiving ASCT; 3-year survival for high-risk with or without ASCT was 42% versus 0% (p < 0.01); intermediate 72% versus 43% (p = 0.06); and low-risk 88% versus 78% (p = 0.53). The Mayo genetic risk models offer pre-treatment and response-based prognostic tools for ND-AML treated with Ven-HMA. The current study underscores the prognostically indispensable role of achieving CR/CRi and ASCT.
{"title":"Mayo Genetic Risk Models for Newly Diagnosed Acute Myeloid Leukemia Treated With Venetoclax + Hypomethylating Agent","authors":"Naseema Gangat, Azeem Elbeih, Nour Ghosoun, Kristen McCullough, Fnu Aperna, Isla M. Johnson, Maymona Abdelmagid, Aref Al-Kali, Hassan B. Alkhateeb, Kebede H. Begna, Michelle Elliott, Abhishek Mangaonkar, Aasiya Matin, Antoine N. Saliba, Mehrdad Hefazi Torghabeh, Mark R. Litzow, William Hogan, Mithun Shah, Mrinal M. Patnaik, Animesh Pardanani, Talha Badar, Hemant Murthy, James Foran, Jeanne Palmer, Lisa Sproat, Nandita Khera, Cecilia Arana Yi, Samuel Yates, Abigail Sneider, Emily Dworkin, Anand A. Patel, Alexandre Bazinet, Jayastu Senapati, Alex Bataller, Courtney DiNardo, Tapan Kadia, Ayalew Tefferi","doi":"10.1002/ajh.27564","DOIUrl":"https://doi.org/10.1002/ajh.27564","url":null,"abstract":"Patients with newly diagnosed acute myeloid leukemia (ND-AML) derive variable survival benefit from venetoclax + hypomethylating agent (Ven-HMA) therapy. The primary objective in the current study was to develop genetic risk models that are predictive of survival and are applicable at the time of diagnosis and after establishing treatment response. Among 400 ND-AML patients treated with Ven-HMA at the Mayo Clinic, 247 (62%) achieved complete remission with (CR) or without (CRi) count recovery. Multivariable analysis–derived hazard ratios (HR), including 1.8 for European LeukemiaNet (ELN) adverse karyotype, 4.7 for <i>KMT2Ar</i>, 1.7 for <i>TP53</i><sup>MUT</sup>, 2.6 for <i>KRAS</i> <sup>MUT</sup>, and 2.1 for <i>IDH2</i><sup>WT</sup> were applied to develop an HR-weighted risk model: low, intermediate, and high; respective median survival censored for allogeneic stem cell transplant (ASCT) (3-year survival) were “not reached” (67%), 19.1 (33%), and 7.1 months (0%). In patients achieving CR/CRi, adverse karyotype, <i>KMT2Ar</i>, <i>KRAS</i><sup>MUT</sup>, <i>IDH2</i><sup>WT</sup> predicted inferior survival, allowing for a complementary response-stratified risk model. The model was externally validated and was shown to be superior to the ELN 2024 risk model (AIC 179 vs. 195 and AUC 0.77 vs. 0.69). Survival was inferior with failure to achieve CR/CRi or not receiving ASCT; 3-year survival for high-risk with or without ASCT was 42% versus 0% (<i>p</i> < 0.01); intermediate 72% versus 43% (<i>p</i> = 0.06); and low-risk 88% versus 78% (<i>p</i> = 0.53). The Mayo genetic risk models offer pre-treatment and response-based prognostic tools for ND-AML treated with Ven-HMA. The current study underscores the prognostically indispensable role of achieving CR/CRi and ASCT.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"12 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142816212","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Petra Stute, Imo J. Akpan, Christian Breymann, Ally Murji, Sarah H. O'Brien, Jacquelyn M. Powers, Malcolm G. Munro
<p>Anemia is prevalent among women of reproductive age, with iron deficiency (ID) being the primary etiology. ID can lead to fatigue and reduced quality of life, particularly in the context of abnormal menstrual bleeding [<span>1</span>]. Abnormal menstrual bleeding refers to a set of symptoms collectively known as abnormal uterine bleeding (AUB), and includes heavy menstrual bleeding (HMB), experienced by up to around 50% of reproductive-aged women [<span>1, 2</span>]. Notably, as many as 63% of women with HMB have been reported to have ID or iron deficiency anemia (IDA) [<span>1</span>]. Despite symptoms of IDA being common alongside AUB, there is a paucity of data on response to intravenous (IV) iron treatment in such a population.</p> <p>This <i>post hoc</i> analysis evaluated pooled data from IV iron-treated participants with AUB-associated IDA from two Phase III trials, PROVIDE and FERWON-IDA (ClinicalTrials.gov identifiers: NCT02130063 and NCT02940886, respectively). The detailed study designs and results of these trials have been published previously [<span>3, 4</span>]. While the trials compared the efficacy and safety of ferric derisomaltose (FDI; a high-dose formulation) to a low-dose IV iron, iron sucrose (IS), the main focus of this analysis was the effect of FDI – the newer IV iron associated with increased convenience through its high-dose regimen – on fatigue, a symptom frequently experienced by women with ID or IDA [<span>1</span>].</p> <p>Female participants from the FDI treatment arms of PROVIDE and FERWON-IDA were included in this <i>post hoc</i> analysis if they had received treatment, were aged 18–55 years, and had AUB-associated IDA (defined as hemoglobin [Hb] < 12 g/dL, serum ferritin < 100 ng/mL, and transferrin saturation < 20% at baseline). The underlying cause of IDA was recorded in the trials, but AUB was not a term specified in the case report form; instead, a range of terms were used, including menorrhagia, metrorrhagia, and uterine hemorrhage, which were categorized under AUB in this <i>post hoc</i> analysis. Participants with uterine leiomyoma listed as the cause of their IDA were also included in the analysis, as it was assumed that they experienced AUB.</p> <p>The primary outcome was the change from Week 0 (baseline) to Week 8 (last study visit in FERWON-IDA) in the Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale score, a 13-item patient-reported measure of fatigue and its impact on daily functioning over the past 7 days [<span>5</span>]. Each item is marked from 0 (“not at all”) to 4 (“very much”) on a 5-point Likert scale [<span>5</span>]. The data are then transformed according to scoring guidelines [<span>5</span>]. The resulting total score falls within the range of 0–52, with a lower score indicating greater fatigue [<span>5</span>]. Fatigue data were collected by both trials.</p> <p>Other outcomes included change from baseline to Week 8 in Hb, and the proportions of partici
{"title":"Effect of Ferric Derisomaltose on Fatigue in Iron Deficiency Anemia Associated With Abnormal Uterine Bleeding","authors":"Petra Stute, Imo J. Akpan, Christian Breymann, Ally Murji, Sarah H. O'Brien, Jacquelyn M. Powers, Malcolm G. Munro","doi":"10.1002/ajh.27555","DOIUrl":"https://doi.org/10.1002/ajh.27555","url":null,"abstract":"<p>Anemia is prevalent among women of reproductive age, with iron deficiency (ID) being the primary etiology. ID can lead to fatigue and reduced quality of life, particularly in the context of abnormal menstrual bleeding [<span>1</span>]. Abnormal menstrual bleeding refers to a set of symptoms collectively known as abnormal uterine bleeding (AUB), and includes heavy menstrual bleeding (HMB), experienced by up to around 50% of reproductive-aged women [<span>1, 2</span>]. Notably, as many as 63% of women with HMB have been reported to have ID or iron deficiency anemia (IDA) [<span>1</span>]. Despite symptoms of IDA being common alongside AUB, there is a paucity of data on response to intravenous (IV) iron treatment in such a population.</p>\u0000<p>This <i>post hoc</i> analysis evaluated pooled data from IV iron-treated participants with AUB-associated IDA from two Phase III trials, PROVIDE and FERWON-IDA (ClinicalTrials.gov identifiers: NCT02130063 and NCT02940886, respectively). The detailed study designs and results of these trials have been published previously [<span>3, 4</span>]. While the trials compared the efficacy and safety of ferric derisomaltose (FDI; a high-dose formulation) to a low-dose IV iron, iron sucrose (IS), the main focus of this analysis was the effect of FDI – the newer IV iron associated with increased convenience through its high-dose regimen – on fatigue, a symptom frequently experienced by women with ID or IDA [<span>1</span>].</p>\u0000<p>Female participants from the FDI treatment arms of PROVIDE and FERWON-IDA were included in this <i>post hoc</i> analysis if they had received treatment, were aged 18–55 years, and had AUB-associated IDA (defined as hemoglobin [Hb] < 12 g/dL, serum ferritin < 100 ng/mL, and transferrin saturation < 20% at baseline). The underlying cause of IDA was recorded in the trials, but AUB was not a term specified in the case report form; instead, a range of terms were used, including menorrhagia, metrorrhagia, and uterine hemorrhage, which were categorized under AUB in this <i>post hoc</i> analysis. Participants with uterine leiomyoma listed as the cause of their IDA were also included in the analysis, as it was assumed that they experienced AUB.</p>\u0000<p>The primary outcome was the change from Week 0 (baseline) to Week 8 (last study visit in FERWON-IDA) in the Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale score, a 13-item patient-reported measure of fatigue and its impact on daily functioning over the past 7 days [<span>5</span>]. Each item is marked from 0 (“not at all”) to 4 (“very much”) on a 5-point Likert scale [<span>5</span>]. The data are then transformed according to scoring guidelines [<span>5</span>]. The resulting total score falls within the range of 0–52, with a lower score indicating greater fatigue [<span>5</span>]. Fatigue data were collected by both trials.</p>\u0000<p>Other outcomes included change from baseline to Week 8 in Hb, and the proportions of partici","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"62 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142810172","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Julia Weisinger, Raïda Bouzid, Jehane Fadlallah, Christelle Barbet, Francois Provot, Pascale Poullin, Antoine Neel, Manon Marie, Virginie Rieu, Tarik Kanouni, Olivier Moranne, Elie Azoulay, Zora Marjanovic, Elise Corre, Anne-Christine Joly, Minh-Tam Baylatry, Bérangère S. Joly, Agnes Veyradier, Paul Coppo
<p>Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare form of thrombotic microangiopathy (TMA) caused by an autoantibody-mediated deficiency of the von Willebrand factor (vWF) cleaving protease ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin-1 motifs, 13th Member) [<span>1</span>]. Without treatment, the disease is almost always fatal. In the last decades, the combination of therapeutic plasma exchange (TPE), corticosteroids, the chimeric anti-CD20 B-cell depleting monoclonal antibody rituximab and the anti-vWF nanobody caplacizumab, resulted in an improvement of survival rate, now exceeding 90% in the acute phase [<span>2, 3</span>]. However, relapses may occur during follow-up, exposing patients to death and treatment-related complications while increasing the burden of care. To circumvent these issues, a regular monitoring of ADAMTS13 activity during follow up, and the use of preemptive rituximab treatment in patients experiencing a severe ADAMTS13 deficiency (ADAMTS13 relapse) became a standard of care [<span>3-5</span>]. However, up to 15% of patients fail to improve ADAMTS13 activity following rituximab treatment; moreover, patients can develop adverse events, including immediate infusion intolerance or later serum sickness. In this context, alternative therapeutic options are needed, while evidence-based experience or direct comparisons of possible agents are scarce.</p> <p>Obinutuzumab (Gazyvaro, Roche) is a type 2 humanized anti-CD20 monoclonal antibody, glyco-engineered to potently induce direct death of CD20-positive cells. Obinutuzumab proved to be efficient in various B-cell malignancies even following previous rituximab treatments, as well as in autoimmune diseases, where rituximab was contraindicated due to either resistance or intolerance. In iTTP, case reports and small series of patients suggested an efficacy for obinutuzumab in patients with intolerance or refractoriness to rituximab [<span>6</span>]. Here, we report from a large series of patients more definitive evidence that obinutuzumab is efficient and safe in preventing relapses in iTTP patients experiencing refractoriness or intolerance to rituximab.</p> <p>Data on patients with a diagnosis of iTTP treated with obinutuzumab in the registry of the French Thrombotic Microangiopathies Reference Center (www.cnr-mat.fr) have been collected according to a predefined computerized dataset. Treatment of iTTP in the acute phase was based on current national and international guidelines [<span>2-5</span>]. Obinutuzumab was used as a compassionate off-label therapy; it was considered in patients with a persistent severe ADAMTS13 deficiency despite the use of rituximab, combined or not with other immunomodulators, or in patients with an ADAMTS13 relapse and intolerance to rituximab. Persistent severe ADAMTS13 deficiency was defined by an ADAMTS13 activity < 20% on at least two consecutive time points, on patients usually assessed for ADAMTS13 ac
11名患者发生了与治疗相关的不良事件（表S4）。最常见的毒性反应是输液相关反应，有6名患者出现了这种反应。从这一大系列患者中，我们提供了更多确凿证据，证明奥比妥珠单抗可使对利妥昔单抗和其他免疫抑制剂无反应或不耐受的iTTP患者获得高应答率。在我们的研究中，启动奥比妥珠单抗治疗的主要原因之一是利妥昔单抗治疗后的血清病。在这种情况下，奥比妥珠单抗对这些患者来说是一种极具吸引力的 B 细胞清除替代策略。虽然也有人描述过使用奥比奴珠单抗后出现血清病的情况，但这种情况似乎很少见；在这方面，我们的研究队列中没有记录到使用奥比奴珠单抗后出现血清病的情况。奥比奴珠单抗对既往对利妥昔单抗耐药的患者也很有效，反应率高达 77%。奥比奴珠单抗规避利妥昔单抗难治性的确切机制尚不完全清楚。在自身免疫性疾病方面的研究强调，奥比妥珠单抗具有卓越的B细胞耗竭能力，这是提高效率的背景。我们研究的局限性包括数据收集的回顾性；虽然法国的TTP一般诊断和治疗指南已完全标准化，但不同中心在开始新的抢先治疗后进行首次ADAMTS13测量的时间点仍不尽相同。据报道，环孢素 A 和环磷酰胺淋巴消融术是很有价值的选择，但它们的耐受性可能会限制其使用。硼替佐米（bortezomib）和达拉曲木单抗（daratumumab）等血浆细胞导向疗法的数据有限，可能只适用于对强化B细胞清除无效的患者。因此，根据我们的研究结果，奥比妥珠单抗可作为对利妥昔单抗不耐受或难治的 iTTP 患者的首选药物。国际前瞻性试验的努力应能更明确地证明奥比妥珠单抗在 iTTP 中作为一种有价值的挽救性免疫抑制疗法的作用。
{"title":"Efficacy and Safety of Obinutuzumab in Immune-Mediated Thrombotic Thrombocytopenic Purpura","authors":"Julia Weisinger, Raïda Bouzid, Jehane Fadlallah, Christelle Barbet, Francois Provot, Pascale Poullin, Antoine Neel, Manon Marie, Virginie Rieu, Tarik Kanouni, Olivier Moranne, Elie Azoulay, Zora Marjanovic, Elise Corre, Anne-Christine Joly, Minh-Tam Baylatry, Bérangère S. Joly, Agnes Veyradier, Paul Coppo","doi":"10.1002/ajh.27550","DOIUrl":"https://doi.org/10.1002/ajh.27550","url":null,"abstract":"<p>Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare form of thrombotic microangiopathy (TMA) caused by an autoantibody-mediated deficiency of the von Willebrand factor (vWF) cleaving protease ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin-1 motifs, 13th Member) [<span>1</span>]. Without treatment, the disease is almost always fatal. In the last decades, the combination of therapeutic plasma exchange (TPE), corticosteroids, the chimeric anti-CD20 B-cell depleting monoclonal antibody rituximab and the anti-vWF nanobody caplacizumab, resulted in an improvement of survival rate, now exceeding 90% in the acute phase [<span>2, 3</span>]. However, relapses may occur during follow-up, exposing patients to death and treatment-related complications while increasing the burden of care. To circumvent these issues, a regular monitoring of ADAMTS13 activity during follow up, and the use of preemptive rituximab treatment in patients experiencing a severe ADAMTS13 deficiency (ADAMTS13 relapse) became a standard of care [<span>3-5</span>]. However, up to 15% of patients fail to improve ADAMTS13 activity following rituximab treatment; moreover, patients can develop adverse events, including immediate infusion intolerance or later serum sickness. In this context, alternative therapeutic options are needed, while evidence-based experience or direct comparisons of possible agents are scarce.</p>\u0000<p>Obinutuzumab (Gazyvaro, Roche) is a type 2 humanized anti-CD20 monoclonal antibody, glyco-engineered to potently induce direct death of CD20-positive cells. Obinutuzumab proved to be efficient in various B-cell malignancies even following previous rituximab treatments, as well as in autoimmune diseases, where rituximab was contraindicated due to either resistance or intolerance. In iTTP, case reports and small series of patients suggested an efficacy for obinutuzumab in patients with intolerance or refractoriness to rituximab [<span>6</span>]. Here, we report from a large series of patients more definitive evidence that obinutuzumab is efficient and safe in preventing relapses in iTTP patients experiencing refractoriness or intolerance to rituximab.</p>\u0000<p>Data on patients with a diagnosis of iTTP treated with obinutuzumab in the registry of the French Thrombotic Microangiopathies Reference Center (www.cnr-mat.fr) have been collected according to a predefined computerized dataset. Treatment of iTTP in the acute phase was based on current national and international guidelines [<span>2-5</span>]. Obinutuzumab was used as a compassionate off-label therapy; it was considered in patients with a persistent severe ADAMTS13 deficiency despite the use of rituximab, combined or not with other immunomodulators, or in patients with an ADAMTS13 relapse and intolerance to rituximab. Persistent severe ADAMTS13 deficiency was defined by an ADAMTS13 activity < 20% on at least two consecutive time points, on patients usually assessed for ADAMTS13 ac","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"14 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142816213","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Lunning MA, Wang HL, Hu ZH, et al., “Benefit of Axicabtagene Ciloleucel Versus Chemoimmunotherapy in Older Patients and/or Patients With Poor ECOG Performance Status With Relapsed or Refractory Large B-Cell Lymphoma After 2 or More Lines of Prior Therapy,” American Journal of Hematology 99, no. 5 (2024): 880–889.
In Figure 1C, a typographical error was made reporting incorrect median overall survival (OS) values for the axi-cel and chemoimmunotherapy (CIT) arms by Eastern Cooperative Oncology Group performance status (ECOG PS). The correct values are reported in Table S2 and are reproduced here:
{"title":"Correction to “Benefit of Axicabtagene Ciloleucel Versus Chemoimmunotherapy in Older Patients and/or Patients With Poor ECOG Performance Status With Relapsed or Refractory Large B-Cell Lymphoma After 2 or More Lines of Prior Therapy”","authors":"","doi":"10.1002/ajh.27551","DOIUrl":"https://doi.org/10.1002/ajh.27551","url":null,"abstract":"<p>Lunning MA, Wang HL, Hu ZH, et al., “Benefit of Axicabtagene Ciloleucel Versus Chemoimmunotherapy in Older Patients and/or Patients With Poor ECOG Performance Status With Relapsed or Refractory Large B-Cell Lymphoma After 2 or More Lines of Prior Therapy,” <i>American Journal of Hematology</i> 99, no. 5 (2024): 880–889.</p>\u0000<p>In Figure 1C, a typographical error was made reporting incorrect median overall survival (OS) values for the axi-cel and chemoimmunotherapy (CIT) arms by Eastern Cooperative Oncology Group performance status (ECOG PS). The correct values are reported in Table S2 and are reproduced here:</p>\u0000<p>Median OS (95% CI).</p>\u0000<p>Axi-cel, ECOG PS < 2 (<i>n</i> = 526): 28.0 months (20.8-NE).</p>\u0000<p>CIT, ECOG PS < 2 (<i>n</i> = 296): 6.4 months (5.7–7.6).</p>\u0000<p>Axi-cel, ECOG PS = 2 (<i>n</i> = 33): 3.6 months (2.3–8.1).</p>\u0000<p>CIT, ECOG PS = 2 (<i>n</i> = 27): 2.7 months (1.4–5.1).</p>\u0000<p>We apologize for this error.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"88 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142810170","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Peri-Transplant Management of JAK Inhibitor Therapy in Myelofibrosis","authors":"Ayalew Tefferi, Vincent T. Ho","doi":"10.1002/ajh.27559","DOIUrl":"https://doi.org/10.1002/ajh.27559","url":null,"abstract":"<h2> Conflicts of Interest</h2>\u0000<p>The authors declare no conflicts of interest.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"42 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142816214","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Roberto Cairoli, Arianna Gatti, Giovanni Grillo, Marta Rachele Stefanucci, Barbara Di Camillo, Monica Fumagalli, Mauro Krampera, Gianpaolo Nadali, Patrizia Zappasodi, Erika Borlenghi, Elisabetta Todisco, Marta Ubezio, Massimo Bernardi, Alfredo Molteni, Claudia Basilico, Mauro Turrini, Rosa Greco, Valentina Mancini, Marta Riva, Davide Paolo Bernasconi, Bruno Brando, Silvio Marco Veronese, Alessandro Beghini
Samples from 34 adult patients newly diagnosed with core binding factor leukemia (CBFL) were collected both at the time of diagnosis and at relapse and were centrally analyzed. Eligible patients received either standard induction CT known as “3 + 7” or an equivalent regimen, according to the recruiting center's policy. Patients who achieved CR or CRi received 3 courses of high-dose ARA-C (Cytarabine) 3000 mg/m2 every 12 h on days 1, 3, and 5, along with midostaurin at the dose of 50 mg b.i.d from Day 8 to Day 21 as part of consolidation therapy. Following the completion of the consolidation phase, patients received midostaurin as a monotherapy at the dose of 50 mg b.i.d. for 1 year as continuation therapy. The CR rate was 97%; we recorded an OS rate of 73.52% and a DFS rate of 48.4% for the entire cohort. The RI was 38.8% in the CBFB::MYH11 and 66.6% in the RUNX1::RUNX1T1 group. MRD (Measurable Residual Disease) was assessed by RQ-PCR at 10 time points throughout the study, as indicated by arrows.
{"title":"Efficacy of Midostaurin Combined With Intensive Chemotherapy in Core Binding Factor Leukemia: A Phase II Clinical Trial","authors":"Roberto Cairoli, Arianna Gatti, Giovanni Grillo, Marta Rachele Stefanucci, Barbara Di Camillo, Monica Fumagalli, Mauro Krampera, Gianpaolo Nadali, Patrizia Zappasodi, Erika Borlenghi, Elisabetta Todisco, Marta Ubezio, Massimo Bernardi, Alfredo Molteni, Claudia Basilico, Mauro Turrini, Rosa Greco, Valentina Mancini, Marta Riva, Davide Paolo Bernasconi, Bruno Brando, Silvio Marco Veronese, Alessandro Beghini","doi":"10.1002/ajh.27547","DOIUrl":"https://doi.org/10.1002/ajh.27547","url":null,"abstract":"Samples from 34 adult patients newly diagnosed with core binding factor leukemia (CBFL) were collected both at the time of diagnosis and at relapse and were centrally analyzed. Eligible patients received either standard induction CT known as “3 + 7” or an equivalent regimen, according to the recruiting center's policy. Patients who achieved CR or CRi received 3 courses of high-dose ARA-C (Cytarabine) 3000 mg/m<sup>2</sup> every 12 h on days 1, 3, and 5, along with midostaurin at the dose of 50 mg b.i.d from Day 8 to Day 21 as part of consolidation therapy. Following the completion of the consolidation phase, patients received midostaurin as a monotherapy at the dose of 50 mg b.i.d. for 1 year as continuation therapy. The CR rate was 97%; we recorded an OS rate of 73.52% and a DFS rate of 48.4% for the entire cohort. The RI was 38.8% in the <i>CBFB::MYH11</i> and 66.6% in the <i>RUNX1::RUNX1T1</i> group. MRD (Measurable Residual Disease) was assessed by RQ-PCR at 10 time points throughout the study, as indicated by arrows.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"19 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142804733","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Spiculated erythrocytes come in a variety of forms, which are of diagnostic importance. They can be broadly classified as echinocytes, acanthocytes, keratocytes, and schistocytes. However, complexity is added by the superimposition of one abnormality on another. Other spiculated cells can, for example, undergo an echinocytic or acanthocytic change. In addition, other poikilocytes, for example, spherocytes, can also undergo spiculation.
京公网安备 11010802042870号
京ICP备2023020795号-1
扫码关注我们
求助内容：
应助结果提醒方式：