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Impact of Rituximab Maintenance on outcomes in Follicular Lymphoma: An indian experience 利妥昔单抗维持对滤泡性淋巴瘤预后的影响:印度经验
IF 0.9 Q4 HEMATOLOGY Pub Date : 2025-11-29 DOI: 10.1016/j.lrr.2025.100554
Harish Pant , Ajay Gogia , Atul Sharma , Naveet Wig , Hari Krishna Raju Sagiraju , Saumyaranjan Mallick , Ritu Gupta , Ahitagni Biswas

Background

Follicular lymphoma (FL) is the second most common subtype of non-Hodgkin lymphoma (NHL) and comprises 9 % of total NHL in India. Rituximab maintenance (RM) after induction immunochemotherapy improves PFS. However, currently, no data exists from the Indian continent regarding the impact of Rituximab maintenance (RM) on overall survival (OS) & progression-free survival (PFS). Our study aims to assess if RM improves the outcomes in FL patients.

Methods

This retrospective study included 95 patients diagnosed with FL meeting GELF criteria and registered at our institute All India Institute of Medical Sciences Delhi between January 2012 -December 2023. Among these, forty -four (46 %) patients received 2 years of RM every 2 months after induction immunochemotherapy. The primary outcome were overall survival (OS) and progression-free survival. Secondary outcomes were factors affecting OS & PFS.

Results

Out of the 95 patients who met GELF criteria, 52 were male and 43 were female, with a median age of 53 years (range: 27–81). Advanced Ann Arbor stage III/IV comprises 82 (86 %) patients. Bone marrow involvement and bulky disease were observed in 36 (38 %) and 29 (31 %) patients respectively. Based on the Follicular Lymphoma International Prognostic Index (FLIPI-1), 27 (28 %) were low risk, 9 (10 %) intermediate risk, and 59 (62 %) were high risk. The complete remission (CR) was achieved in 71 % of patients. During follow-up, 27 (28 %) patients experienced relapsed and 8 patients transformed to diffuse large B-cell lymphoma (DLBCL). At a median follow-up of 63 months, the median OS was not reached. The median PFS was 122 months in the RM group and 94 months in the non-RM group, with 5-year PFS of 91.6 % vs 59.3 %, respectively (log-rank p = 0.017). Rituximab maintenance was independently associated with improved PFS (adjusted HR 0.28, 95 % CI: 0.12–0.65, p = 0.003). Failure to attain CR (aHR 2.49, 95 % CI: 1.20–5.19, p = 0.015) and bone marrow involvement (aHR 2.92, 95 % CI: 0.99–8.63, p = 0.05) were independently associated with inferior PFS.

Conclusions

Rituximab maintenance after induction immunochemotherapy significantly improved PFS. This is the first study from India demonstrating the impact of rituximab maintenance in FL.
滤泡性淋巴瘤(FL)是第二常见的非霍奇金淋巴瘤(NHL)亚型,占印度NHL总数的9%。诱导免疫化疗后利妥昔单抗维持(RM)改善PFS。然而,目前还没有来自印度大陆的关于利妥昔单抗维持(RM)对总生存期(OS)和无进展生存期(PFS)影响的数据。我们的研究旨在评估RM是否能改善FL患者的预后。方法回顾性研究纳入2012年1月至2023年12月在我们研究所全印度医学科学研究所登记的95例符合GELF标准的FL患者。其中,44例(46%)患者在诱导免疫化疗后每2个月接受2年的化疗。主要终点是总生存期(OS)和无进展生存期。次要结局是影响OS和PFS的因素。结果95例符合GELF标准的患者中,男性52例,女性43例,中位年龄53岁(范围:27-81岁)。晚期安娜堡III/IV期包括82例(86%)患者。骨髓受累36例(38%),大面积病变29例(31%)。根据滤泡性淋巴瘤国际预后指数(FLIPI-1), 27例(28%)为低危,9例(10%)为中危,59例(62%)为高危。71%的患者达到完全缓解(CR)。在随访期间,27例(28%)患者复发,8例转化为弥漫性大b细胞淋巴瘤(DLBCL)。在中位随访63个月时,中位OS未达到。RM组和非RM组的中位PFS分别为122个月和94个月,5年PFS分别为91.6%和59.3% (log-rank p = 0.017)。利妥昔单抗维持与PFS改善独立相关(调整后危险度0.28,95% CI: 0.12-0.65, p = 0.003)。未能达到CR (aHR 2.49, 95% CI: 1.20 ~ 5.19, p = 0.015)和骨髓受累(aHR 2.92, 95% CI: 0.99 ~ 8.63, p = 0.05)与PFS较差独立相关。结论诱导免疫化疗后维持斯利妥昔单抗可显著改善PFS。这是印度第一个证明利妥昔单抗维持对FL影响的研究。
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引用次数: 0
A rare case of variant acute promyelocytic leukemia with FIP1L1-RARA fusion gene: case report and literature review 罕见的FIP1L1-RARA融合基因变异型急性早幼粒细胞白血病1例报告并文献复习
IF 0.9 Q4 HEMATOLOGY Pub Date : 2025-11-25 DOI: 10.1016/j.lrr.2025.100553
Yanyan Qiu, Huarong Zhou, Shaoyuan Wang
Acute promyelocytic leukemia (APL) is a distinct subtype of acute myeloid leukemia characterized by the promyelocytic leukemia-retinoic acid receptor alpha (PML-RARA) fusion gene and exceptional responsiveness to differentiation therapy with all-trans retinoic acid (ATRA) and arsenic trioxide (Arsenic Trioxide). However, rare variant forms of APL involving alternative RARA fusion partners, such as FIP1L1-RARA, are typically resistant to ATRA/ATO-based regimens and are associated with poor clinical outcomes. We report a rare case of FIP1L1-RARA-positive APL in a patient who initially presented with clinical, morphological, and immunophenotypic features consistent with classical APL. Standard diagnostic evaluations, including morphology, flow cytometry, and fluorescence in situ hybridization (FISH), failed to detect the fusion abnormality. The diagnosis was ultimately confirmed by RNA sequencing (RNA-Seq). Empirical induction with ATRA and ATO was initiated but resulted in persistent and progressive promyelocytosis. Anthracycline-based chemotherapy was subsequently administered, followed by azacitidine combined with venetoclax in the subsequent course of treatment. Although partial hematologic improvement was observed, the overall response remained suboptimal. This case underscores the diagnostic challenges of atypical APL and highlights the pivotal role of RNA-Seq in identifying cryptic RARA rearrangements. A literature review suggests that FIP1L1-RARA-positive APL represents a biologically and clinically distinct entity with highly variable treatment responses and poor prognosis. Early molecular diagnosis and prompt implementation of conventional chemotherapy or targeted therapies such as venetoclax may be essential to improving outcomes in this rare APL subtype.
急性早幼粒细胞白血病(APL)是一种独特的急性髓性白血病亚型,其特征是早幼粒细胞白血病-视黄酸受体α (PML-RARA)融合基因,以及对全反式视黄酸(ATRA)和三氧化二砷(砷三氧化二砷)分化治疗的特殊反应。然而,涉及替代RARA融合伙伴的罕见变异形式的APL,如FIP1L1-RARA,通常对基于ATRA/ ato的方案具有耐药性,并且与较差的临床结果相关。我们报告一例罕见的fip1l1 - rara阳性APL患者,其最初表现为与经典APL一致的临床、形态学和免疫表型特征。包括形态学、流式细胞术和荧光原位杂交(FISH)在内的标准诊断评估未能检测到融合异常。最终通过RNA测序(RNA- seq)确诊。ATRA和ATO的经验诱导开始,但导致持续和进行性早幼粒细胞增多症。随后给予蒽环类化疗,随后在随后的治疗过程中给予阿扎胞苷联合venetoclax。虽然观察到部分血液学改善,但总体反应仍然不理想。该病例强调了非典型APL的诊断挑战,并强调了RNA-Seq在识别隐蔽性RARA重排中的关键作用。一项文献综述表明,fip1l1 - rara阳性APL是一种生物学和临床独特的实体,其治疗反应高度可变,预后不良。早期分子诊断和及时实施常规化疗或靶向治疗(如venetoclax)可能对改善这种罕见APL亚型的预后至关重要。
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引用次数: 0
Mechanisms and signaling pathways of tyrosine kinase inhibitor resistance in chronic myeloid leukemia: A comprehensive review. 慢性髓性白血病中酪氨酸激酶抑制剂耐药的机制和信号通路综述。
IF 0.9 Q4 HEMATOLOGY Pub Date : 2025-08-05 eCollection Date: 2025-01-01 DOI: 10.1016/j.lrr.2025.100533
Meriem Lahmouad, Zahrae Rachid, Rawane Bellemrrabet, Jihane Zerrouk, Khan Wen Goh, Abdelhakim Bouyahya, Youssef Aboussalah

Chronic Myeloid Leukemia (CML) is characterized by aberrant BCR::ABL1 tyrosine kinase activity in hematopoietic stem cells. Although tyrosine kinase inhibitors (TKIs) have revolutionized CML treatment, resistance remains a major clinical challenge. This review provides a comprehensive overview of CML, including its epidemiology, pathophysiology, diagnosis, and treatment, as outlined in the latest WHO consensus classification. Current treatment paradigms and the prospects for treatment-free remission (TFR) are also explored. The primary focus is on elucidating the molecular mechanisms of TKI resistance, emphasizing both well-known pathways such as PI3K/AKT, MAPK, JAK/STAT, and alternative pathways including SRC/AKT. This review stands out by integrating recent discoveries regarding genetic mutations within the BCR::ABL1 gene, alongside other molecular alterations contributing to resistance. By synthesizing this knowledge, it aims to guide clinical practitioners, investigators, and translational researchers in developing innovative strategies to overcome resistance and improve patient outcomes in CML.

慢性髓系白血病(CML)以造血干细胞中BCR::ABL1酪氨酸激酶活性异常为特征。虽然酪氨酸激酶抑制剂(TKIs)已经彻底改变了CML的治疗,但耐药性仍然是一个主要的临床挑战。本综述提供了CML的全面概述,包括其流行病学、病理生理学、诊断和治疗,概述了最新的世卫组织共识分类。目前的治疗模式和前景的无治疗缓解(TFR)也进行了探讨。主要重点是阐明TKI耐药的分子机制,强调PI3K/AKT、MAPK、JAK/STAT等众所周知的途径,以及包括SRC/AKT在内的替代途径。这篇综述通过整合最近关于BCR::ABL1基因突变的发现,以及其他有助于耐药的分子改变,脱颖而出。通过综合这些知识,它旨在指导临床从业者,研究者和转化研究人员开发创新策略来克服耐药性并改善CML患者的预后。
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引用次数: 0
Characteristics of BCR::ABL1 kinase domain mutations in Vietnamese chronic myeloid leukemia patients 越南慢性髓性白血病患者BCR::ABL1激酶结构域突变特征
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 DOI: 10.1016/j.lrr.2025.100512
Phu Chi Dung , Huynh Duc Vinh Phu , Cao Van Dong , Chau Thuy Ha , Nguyen Thi Thanh Ha , Tran Ngoc Xuan Thy , Le Vu Ha Thanh , Huynh Nghia , Nguyen Tan Binh , Hoang Anh Vu , Phan Thi Xinh , Cao Sy Luan

Background

BCR::ABL1 kinase domain (KD) mutations represent a common cause of resistance to tyrosine kinase inhibitors in chronic myeloid leukemia (CML) patients. The frequency and pattern of KD mutations differ among populations worldwide. However, the characteristics of KD mutations in Vietnamese patients remain unclear.

Methods

A retrospective cohort study of CML patients at Blood Transfusion Hematology Hospital who were resistant to frontline imatinib between Oct 2010 and Oct 2018. Direct sequencing technique was performed to detect KD mutations.

Results

488 imatinib-resistant CML patients were included in our study. The median age of the patients was 39, with the majority (82.1 %) diagnosed with chronic phase at the time of resistance. KD mutations were identified in 173 (35.5 %) patients, with 8 cases involving novel variants. The KD mutations predominantly localized within the P-loop of BCR::ABL1 (36.7 %). G250E was the most common mutation, followed by Y253H, M351T, and M244V. In particular, Y253H, T315I, F359V, F317L, E355G, and Q252H were frequently observed in accelerated phase and blast crisis patients. In addition, M244V, T315I, E459K, E255K, F317L, Q252H and E355G were all observed in primary resistant patients.

Conclusion

The emergence of certain specific mutations may serve as the early indicators of leukemic progression, necessitating prompt intervention for better disease control.
dbcr:ABL1激酶结构域(KD)突变是慢性髓性白血病(CML)患者对酪氨酸激酶抑制剂耐药的常见原因。KD突变的频率和模式在世界各地的人群中有所不同。然而,越南患者KD突变的特征尚不清楚。方法对2010年10月至2018年10月输血血液科医院一线伊马替尼耐药CML患者进行回顾性队列研究。采用直接测序技术检测KD突变。结果共纳入488例伊马替尼耐药CML患者。患者的中位年龄为39岁,大多数(82.1%)在耐药时诊断为慢性期。在173例(35.5%)患者中发现KD突变,其中8例涉及新变体。KD突变主要发生在BCR::ABL1的p环内(36.7%)。G250E是最常见的突变,其次是Y253H、M351T和M244V。其中Y253H、T315I、F359V、F317L、E355G、Q252H在加速期和爆炸危象患者中较多出现。此外,M244V、T315I、E459K、E255K、F317L、Q252H和E355G均出现在原发性耐药患者中。结论某些特异性突变的出现可能是白血病进展的早期指标,需要及时干预以更好地控制疾病。
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引用次数: 0
A case report of vanishing bile duct syndrome and diffuse large b cell lymphoma: An uncommon association 胆管消失综合征合并弥漫性大b细胞淋巴瘤1例:罕见关联
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 DOI: 10.1016/j.lrr.2025.100511
Cecilia Anna Fidanza , Ramona Cassin , Francesca Cavallaro , Giorgio Alberto Croci , Francesca Gaia Rossi Dardanoni , Wilma Barcellini , Francesco Passamonti
We report a rare case of vanishing bile duct syndrome (VBDS) associated with diffuse large B cell lymphoma (DLBCL). VBDS is an uncommon ductopenic disorder associated with various underlying conditions, for which timely treatment is crucial to prevent poor outcomes. Our patient received six cycles of R-CHOP chemotherapy every 21 days with halved doxorubicin and vincristine doses, and prophylactic intrathecal chemotherapy. A complete response of DLBCL and normalization of liver parameters were achieved upon completion of treatment. We conclude that, when VBDS is diagnosed, one can consider to urgently reach lymphoma remission even balancing the dose reduction of hepatotoxic drugs.
我们报告一例罕见的胆管消失综合征(VBDS)合并弥漫性大B细胞淋巴瘤(DLBCL)。VBDS是一种罕见的导管减少性疾病,与多种潜在疾病相关,及时治疗对于预防不良后果至关重要。我们的患者接受了6个周期的R-CHOP化疗,每21天使用一半剂量的阿霉素和长春新碱,以及预防性鞘内化疗。治疗结束后,DLBCL完全缓解,肝脏参数正常化。我们的结论是,当诊断出VBDS时,即使平衡肝毒性药物的剂量减少,也可以考虑紧急达到淋巴瘤缓解。
{"title":"A case report of vanishing bile duct syndrome and diffuse large b cell lymphoma: An uncommon association","authors":"Cecilia Anna Fidanza ,&nbsp;Ramona Cassin ,&nbsp;Francesca Cavallaro ,&nbsp;Giorgio Alberto Croci ,&nbsp;Francesca Gaia Rossi Dardanoni ,&nbsp;Wilma Barcellini ,&nbsp;Francesco Passamonti","doi":"10.1016/j.lrr.2025.100511","DOIUrl":"10.1016/j.lrr.2025.100511","url":null,"abstract":"<div><div>We report a rare case of vanishing bile duct syndrome (VBDS) associated with diffuse large B cell lymphoma (DLBCL). VBDS is an uncommon ductopenic disorder associated with various underlying conditions, for which timely treatment is crucial to prevent poor outcomes. Our patient received six cycles of R-CHOP chemotherapy every 21 days with halved doxorubicin and vincristine doses, and prophylactic intrathecal chemotherapy. A complete response of DLBCL and normalization of liver parameters were achieved upon completion of treatment. We conclude that, when VBDS is diagnosed, one can consider to urgently reach lymphoma remission even balancing the dose reduction of hepatotoxic drugs.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"23 ","pages":"Article 100511"},"PeriodicalIF":0.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143878653","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A rare presentation of multiple myeloma with concurrent paraskeletal extramedullary thoracic plasmacytoma: A case report 多发性骨髓瘤并发副骨骼髓外胸椎浆细胞瘤一例报告
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 DOI: 10.1016/j.lrr.2025.100501
Saja I. AbuGhannam , Celina R. Andonie , Yousef Abu Asbeh , Aliaa’ Khalili
Multiple myeloma is a hematological malignancy that results from the proliferation of abnormal plasma cells, typically invading the bone marrow but occasionally involving other areas of the body. We present a rare case of a 58-year-old male patient who presented with right-sided thoracic wall mass, which was eventually diagnosed through imaging and biopsy as paraskeletal extramedullary plasmacytoma with concurrent multiple myeloma. The patient exhibited symptoms of chest pain and swelling, with radiological features of a large right-sided chest wall mass. The diagnosis shows the fact that radiological presentations are quite nonspecific, often mimicking other malignancies. Such cases, therefore, require further assistance from thoracic surgery and interventional radiology in addition to advanced imaging techniques such as FDG- PET. He was subsequently treated with a three-drug regimen (VTD-Zometa protocol) including velcade, thalidomide, and dexamethasone, considering radiation versus complete surgical exicion due to the size of the mass. This case also supports the idea that clinical diversity exists among multiple myeloma and that considering paraskeletal extramedullary plasmacytoma must be given due importance when dealing with differential diagnosis of chest wall tumors in old age, although extremely rare. Early diagnosis and exclusion of all other possible diagnoses are also critical for the best possible treatment planning and outcome.
多发性骨髓瘤是一种由异常浆细胞增殖引起的血液系统恶性肿瘤,通常侵袭骨髓,但偶尔也会累及身体的其他部位。我们报告一例罕见的58岁男性患者,其表现为右侧胸壁肿块,最终通过影像学和活检诊断为副骨骼髓外浆细胞瘤合并多发性骨髓瘤。患者表现出胸痛和肿胀的症状,影像学表现为右侧胸壁大肿块。诊断显示放射学表现是非特异性的,通常与其他恶性肿瘤相似。因此,除了FDG- PET等先进成像技术外,此类病例还需要胸外科和介入放射学的进一步帮助。随后,他接受了包括维卡德、沙利度胺和地塞米松在内的三药治疗方案(VTD-Zometa方案),考虑到肿块的大小,是放疗还是完全手术切除。本病例也支持多发性骨髓瘤存在临床多样性的观点,在老年胸壁肿瘤的鉴别诊断中,尽管极为罕见,但必须重视考虑副骨骼髓外浆细胞瘤。早期诊断和排除所有其他可能的诊断对于最佳治疗计划和结果也至关重要。
{"title":"A rare presentation of multiple myeloma with concurrent paraskeletal extramedullary thoracic plasmacytoma: A case report","authors":"Saja I. AbuGhannam ,&nbsp;Celina R. Andonie ,&nbsp;Yousef Abu Asbeh ,&nbsp;Aliaa’ Khalili","doi":"10.1016/j.lrr.2025.100501","DOIUrl":"10.1016/j.lrr.2025.100501","url":null,"abstract":"<div><div>Multiple myeloma is a hematological malignancy that results from the proliferation of abnormal plasma cells, typically invading the bone marrow but occasionally involving other areas of the body. We present a rare case of a 58-year-old male patient who presented with right-sided thoracic wall mass, which was eventually diagnosed through imaging and biopsy as paraskeletal extramedullary plasmacytoma with concurrent multiple myeloma. The patient exhibited symptoms of chest pain and swelling, with radiological features of a large right-sided chest wall mass. The diagnosis shows the fact that radiological presentations are quite nonspecific, often mimicking other malignancies. Such cases, therefore, require further assistance from thoracic surgery and interventional radiology in addition to advanced imaging techniques such as FDG- PET. He was subsequently treated with a three-drug regimen (VTD-Zometa protocol) including velcade, thalidomide, and dexamethasone, considering radiation versus complete surgical exicion due to the size of the mass. This case also supports the idea that clinical diversity exists among multiple myeloma and that considering paraskeletal extramedullary plasmacytoma must be given due importance when dealing with differential diagnosis of chest wall tumors in old age, although extremely rare. Early diagnosis and exclusion of all other possible diagnoses are also critical for the best possible treatment planning and outcome.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"23 ","pages":"Article 100501"},"PeriodicalIF":0.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143453726","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Clonal eosinophilia with exclusive pulmonary involvement driven by PDGFRA rearrangement treated with imatinib: A case report 伊马替尼治疗PDGFRA重排引起的克隆性嗜酸性粒细胞增多伴排他性肺部受累1例
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 DOI: 10.1016/j.lrr.2025.100502
Zaineb Mlayah, Inés Ben-Rekaya, Inaam Bizid, Nader Slama, Sara Boukhris, Mohamed-Adnene Laatiri
Hypereosinophilic syndrome (HES) was first described in 1968 by Hardy and Anderson. It is a group of rare, multisystemic and heterogeneous pathologies, characterized by significant morbidity and mortality. The occurrence of clonal hypereosinophilic syndrome associated with FIP 1L1-PDGFRA+ is estimated to range between 0.31 and 6.3 cases per million individuals. The organs most commonly impacted are the heart and spleen, with the lungs being the next most affected. Clonal hypereosinophilic syndromes with exclusive pulmonary involvement are exceptional. Due to the rarity of clonal HES, this report aims to not only describe the patient's clinical, biological, and radiological manifestations of clonal HES but also enrich the literature to ameliorate the management of this uncommon syndrome.
we report the case of a patient with past medical history of obstructive bronchopneumopathy who was presented with cough and dyspnea. Investigations revealed peripheral blood hypereosinophilia (between 4000 and 9000/mm3) which lead us to suspect clonal hypereosinophilic syndrome (HES). This diagnosis was confirmed by cytogenetics/fluorescence in situ hybridization (FISH) which demonstrated a positive FIP 1L1-PDGFRA rearrangement. The CTAP confirmed isolated lung involvement with interstitial infiltrate of the subpleural territories of both lung bases and the bronchoalveolar lavage showed eosinophil count elevated at 15%. The patient was treated by imatinib at a dose of 100 mg/day was initiated. The patient follow-up showed a reduction in eosinophils count to 7500/mm3 at two months of treatment. A molecular evaluation is scheduled in 3 months to assess the response to imatinib.
高嗜酸性粒细胞综合征(HES)在1968年由Hardy和Anderson首次描述。它是一组罕见的、多系统的、异质性的病理,以显著的发病率和死亡率为特征。与FIP 1L1-PDGFRA+相关的克隆性高嗜酸性粒细胞综合征的发生率估计在每百万人0.31至6.3例之间。最常受影响的器官是心脏和脾脏,其次是肺部。克隆性高嗜酸性粒细胞综合征只累及肺部是例外。由于克隆性HES罕见,本报告旨在描述克隆性HES患者的临床、生物学和放射学表现,同时丰富文献以改善这种罕见综合征的治疗。我们报告的情况下,病人过去的病史阻塞性支气管肺炎谁是咳嗽和呼吸困难的表现。调查显示外周血嗜酸性粒细胞增多(4000 - 9000/mm3),导致我们怀疑克隆性嗜酸性粒细胞增多综合征(HES)。细胞遗传学/荧光原位杂交(FISH)证实了这一诊断,显示FIP 1L1-PDGFRA重排阳性。CTAP证实孤立性肺受累,双肺基底胸膜下区域间质浸润,支气管肺泡灌洗显示嗜酸性粒细胞计数升高15%。患者开始接受伊马替尼治疗,剂量为100mg /天。患者随访显示,治疗两个月后,嗜酸性粒细胞计数降至7500/mm3。计划在3个月内进行分子评估以评估对伊马替尼的反应。
{"title":"Clonal eosinophilia with exclusive pulmonary involvement driven by PDGFRA rearrangement treated with imatinib: A case report","authors":"Zaineb Mlayah,&nbsp;Inés Ben-Rekaya,&nbsp;Inaam Bizid,&nbsp;Nader Slama,&nbsp;Sara Boukhris,&nbsp;Mohamed-Adnene Laatiri","doi":"10.1016/j.lrr.2025.100502","DOIUrl":"10.1016/j.lrr.2025.100502","url":null,"abstract":"<div><div>Hypereosinophilic syndrome (HES) was first described in 1968 by Hardy and Anderson. It is a group of rare, multisystemic and heterogeneous pathologies, characterized by significant morbidity and mortality. The occurrence of clonal hypereosinophilic syndrome associated with FIP 1L1-PDGFRA+ is estimated to range between 0.31 and 6.3 cases per million individuals. The organs most commonly impacted are the heart and spleen, with the lungs being the next most affected. Clonal hypereosinophilic syndromes with exclusive pulmonary involvement are exceptional. Due to the rarity of clonal HES, this report aims to not only describe the patient's clinical, biological, and radiological manifestations of clonal HES but also enrich the literature to ameliorate the management of this uncommon syndrome.</div><div>we report the case of a patient with past medical history of obstructive bronchopneumopathy who was presented with cough and dyspnea. Investigations revealed peripheral blood hypereosinophilia (between 4000 and 9000/mm3) which lead us to suspect clonal hypereosinophilic syndrome (HES). This diagnosis was confirmed by cytogenetics/fluorescence in situ hybridization (FISH) which demonstrated a positive FIP 1L1-PDGFRA rearrangement. The CTAP confirmed isolated lung involvement with interstitial infiltrate of the subpleural territories of both lung bases and the bronchoalveolar lavage showed eosinophil count elevated at 15%. The patient was treated by imatinib at a dose of 100 mg/day was initiated. The patient follow-up showed a reduction in eosinophils count to 7500/mm3 at two months of treatment. A molecular evaluation is scheduled in 3 months to assess the response to imatinib.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"23 ","pages":"Article 100502"},"PeriodicalIF":0.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143550306","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
FLT3 and NPM1 mRNA expression-based risk stratification of de novo acute Myeloid Leukemia 基于FLT3和NPM1 mRNA表达的新发急性髓系白血病风险分层。
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 DOI: 10.1016/j.lrr.2024.100494
Donghyun Kim , Grerk Sutamtewagul , Yeonhwa Yu
Prognostication of acute myeloid leukemia (AML) at initial diagnosis relies on identification of pre-determined underlying genetic abnormalities. Nevertheless, the disease course of AML remains highly unpredictable and robust reliable prognostic biomarkers for newly diagnosed AML are lacking. We retrospectively explored two publicly available AML RNA-Seq datasets and found that inferior overall survival was associated with high-FLT3 and low-NPM1 transcript levels (“FLT3high/NPM1low”) compared to low-FLT3 and high-NPM1 transcript levels (“FLT3low/NPM1high”) in adult de novo AML patients, with a hazard ratio for death of at least 2. Transcript level-dependent differential overall survival was independent from the underlying FLT3 or NPM1 genotypes. Our two-gene RNA expression-based de novo AML risk stratification may supplement and fine-tune traditional genetic aberration-based prognostication methods.
急性髓性白血病(AML)的早期诊断依赖于预先确定的潜在遗传异常的识别。然而,AML的病程仍然是高度不可预测的,对于新诊断的AML缺乏可靠的预后生物标志物。我们回顾性地研究了两个公开可用的AML RNA-Seq数据集,发现在成年新生AML患者中,与低FLT3和高NPM1转录水平(“FLT3高/NPM1低”)相比,低FLT3和高NPM1转录水平(“FLT3低/NPM1高”)相比,较低的总生存率与高FLT3和低NPM1转录水平相关,死亡风险比至少为2。转录水平依赖的差异总生存率与潜在的FLT3或NPM1基因型无关。我们基于双基因RNA表达的新生AML风险分层可以补充和微调传统的基于遗传畸变的预测方法。
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引用次数: 0
Retreatment with brentuximab vedotin for discordant peripheral T-cell lymphomas 布伦妥昔单抗韦多汀治疗不一致周围t细胞淋巴瘤。
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 DOI: 10.1016/j.lrr.2025.100500
Gen Hasegawa , Noriharu Nakagawa , Yoshimichi Ueda , Masahide Yamazaki
Brentuximab vedotin (BV) has demonstrated efficacy against CD30+ peripheral T-cell lymphoma (PTCL). We herein report a case of CD30+ peripheral T-cell lymphoma, not otherwise specified (PTCL-NOS) that emerged one month after completing BV, cyclophosphamide, doxorubicin, and prednisone (BV-CHP) therapy for anaplastic large cell lymphoma (ALCL) and responded to retreatment with BV monotherapy. This case suggests that CD30+ PTCL emerging shortly after BV-CHP therapy may respond to retreatment with BV monotherapy, even if the phenotype differs from the initial diagnosis.
Brentuximab vedotin (BV)已被证明对CD30+外周t细胞淋巴瘤(PTCL)有效。我们在此报告一例CD30+外周t细胞淋巴瘤,非特异性(PTCL-NOS),在完成BV、环磷酰胺、阿霉素和强的松(BV- chp)治疗间变性大细胞淋巴瘤(ALCL)一个月后出现,并对BV单药治疗有反应。该病例表明,在BV- chp治疗后不久出现的CD30+ PTCL可能对BV单药治疗的再治疗有反应,即使表型与最初的诊断不同。
{"title":"Retreatment with brentuximab vedotin for discordant peripheral T-cell lymphomas","authors":"Gen Hasegawa ,&nbsp;Noriharu Nakagawa ,&nbsp;Yoshimichi Ueda ,&nbsp;Masahide Yamazaki","doi":"10.1016/j.lrr.2025.100500","DOIUrl":"10.1016/j.lrr.2025.100500","url":null,"abstract":"<div><div>Brentuximab vedotin (BV) has demonstrated efficacy against CD30<sup>+</sup> peripheral T-cell lymphoma (PTCL). We herein report a case of CD30<sup>+</sup> peripheral T-cell lymphoma, not otherwise specified (PTCL-NOS) that emerged one month after completing BV, cyclophosphamide, doxorubicin, and prednisone (BV-CHP) therapy for anaplastic large cell lymphoma (ALCL) and responded to retreatment with BV monotherapy. This case suggests that CD30<sup>+</sup> PTCL emerging shortly after BV-CHP therapy may respond to retreatment with BV monotherapy, even if the phenotype differs from the initial diagnosis.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"23 ","pages":"Article 100500"},"PeriodicalIF":0.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11786849/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143081026","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Nivolumab for relapsed and refractory classical Hodgkin lymphoma: retrospective single center analysis 尼武单抗治疗复发和难治性经典霍奇金淋巴瘤:回顾性单中心分析
IF 0.7 Q4 HEMATOLOGY Pub Date : 2025-01-01 DOI: 10.1016/j.lrr.2025.100519
Magdalena Witkowska , Mikołaj Malicki , Weronika Marcinkowska , Kacper Kościelny , Adrianna Kowalik , Damian Mikulski , Grzegorz Mirocha
With the introduction of doxorubicin, bleomycin, vinblastine, and dacarbazine (ABVD) and radiation combined, the prognosis of Hodgkin lymphoma (HL) has significantly improved, with 5-year overall survival of around 90 %. While the lymphoma has become highly curable, the side effects of ABVD treatment are dire and warrant continuous review. Immune checkpoint inhibitors, including nivolumab, have demonstrated high therapeutic efficacy in relapsed and refractory HL patients. Nevertheless, despite much data, the therapy duration and long-term efficacy question remains unresolved. In this retrospective study, in a cohort of 10 patients, we observed a high complete response (CR) rate, while during long-term observation, 5 patients relapsed, and 3 had autoimmune treatment-related complications.
随着阿霉素、博来霉素、长春碱、达卡巴嗪(ABVD)联合放疗的引入,霍奇金淋巴瘤(HL)的预后明显改善,5年总生存率约为90%。虽然淋巴瘤已变得高度可治愈,但ABVD治疗的副作用是可怕的,值得持续审查。免疫检查点抑制剂,包括nivolumab,在复发和难治性HL患者中显示出很高的治疗效果。然而,尽管有大量的数据,治疗时间和长期疗效问题仍未解决。在这项回顾性研究中,在10例患者的队列中,我们观察到高完全缓解率(CR),而在长期观察中,5例患者复发,3例出现自身免疫治疗相关并发症。
{"title":"Nivolumab for relapsed and refractory classical Hodgkin lymphoma: retrospective single center analysis","authors":"Magdalena Witkowska ,&nbsp;Mikołaj Malicki ,&nbsp;Weronika Marcinkowska ,&nbsp;Kacper Kościelny ,&nbsp;Adrianna Kowalik ,&nbsp;Damian Mikulski ,&nbsp;Grzegorz Mirocha","doi":"10.1016/j.lrr.2025.100519","DOIUrl":"10.1016/j.lrr.2025.100519","url":null,"abstract":"<div><div>With the introduction of doxorubicin, bleomycin, vinblastine, and dacarbazine (ABVD) and radiation combined, the prognosis of Hodgkin lymphoma (HL) has significantly improved, with 5-year overall survival of around 90 %. While the lymphoma has become highly curable, the side effects of ABVD treatment are dire and warrant continuous review. Immune checkpoint inhibitors, including nivolumab, have demonstrated high therapeutic efficacy in relapsed and refractory HL patients. Nevertheless, despite much data, the therapy duration and long-term efficacy question remains unresolved. In this retrospective study, in a cohort of 10 patients, we observed a high complete response (CR) rate, while during long-term observation, 5 patients relapsed, and 3 had autoimmune treatment-related complications.</div></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"24 ","pages":"Article 100519"},"PeriodicalIF":0.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144481226","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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Leukemia Research Reports
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