Pub Date : 2019-08-01DOI: 10.33590/emjhematol/10313792
Prachi Gandhi, Santosh Kondekar
Neonatal sepsis is a major cause of morbidity and mortality in newborns. It presents a diagnostic challenge to the neonatologists due to a lack of objective evaluation. It may mimic noninfective conditions, such as inborn error of metabolism, birth asphyxia, and even respiratory distress syndrome in preterms. Nonetheless, over-diagnosis and initiating unwanted empirical antibiotics may pose the threat of drug resistance, increasing the hospital stay and cost of treatment. Traditionally, investigations such as white blood cell count, absolute neutrophil count, immature to total neutrophil ratio, C-reactive protein levels, and blood cultures have been used to diagnose sepsis. However, these have low sensitivity and specificity because they may be elevated in conditions other than sepsis. The in-depth understanding of the neonatal immune system’s response to early infection has led to the discovery of advanced diagnostic tools, including biomarkers. This literature review briefs on the various haematological parameters and biomarkers in neonatal sepsis, exploring newer biomarkers and comparing them with their older counterparts. This will help early diagnosis, treatment, and improved prognosis in neonatal sepsis. As there is a spectrum of markers for diagnosing neonatal sepsis, it is preferable to compile these markers and correlate clinically. A thorough search of this literature was done on the electronic databases PubMed, Elsevier’s Web of Science, and the Cochrane Library. The authors found around 90 relevant articles: 84 were from PubMed, 4 from Elsevier, and 2 from the latest Cochrane database. Of these articles, 57 were selected from between early 2000 and January 2019.
新生儿败血症是新生儿发病和死亡的主要原因。由于缺乏客观的评价，对新生儿医生提出了诊断挑战。它可能模仿非传染性疾病，如先天性代谢错误，出生窒息，甚至早产儿呼吸窘迫综合征。然而，过度诊断和使用不必要的经验性抗生素可能会造成耐药性的威胁，增加住院时间和治疗费用。传统上，诸如白细胞计数、绝对中性粒细胞计数、未成熟中性粒细胞与总中性粒细胞的比率、c反应蛋白水平和血液培养等调查已被用于诊断败血症。然而，这些具有较低的敏感性和特异性，因为它们可能在脓毒症以外的条件下升高。新生儿免疫系统对早期感染反应的深入了解导致了包括生物标志物在内的先进诊断工具的发现。这篇文献综述简要介绍了新生儿败血症的各种血液学参数和生物标志物，探索新的生物标志物，并将它们与旧的生物标志物进行比较。这将有助于新生儿败血症的早期诊断、治疗和改善预后。由于诊断新生儿脓毒症有一系列的标记物，因此最好将这些标记物汇编并在临床上进行关联。在PubMed, Elsevier 's Web of Science和Cochrane Library的电子数据库上对这些文献进行了彻底的搜索。作者发现了大约90篇相关文章:84篇来自PubMed, 4篇来自Elsevier, 2篇来自最新的Cochrane数据库。在这些文章中，有57篇选自2000年初至2019年1月。
{"title":"A Review of the Different Haematological Parameters and Biomarkers Used for Diagnosis of Neonatal Sepsis","authors":"Prachi Gandhi, Santosh Kondekar","doi":"10.33590/emjhematol/10313792","DOIUrl":"https://doi.org/10.33590/emjhematol/10313792","url":null,"abstract":"Neonatal sepsis is a major cause of morbidity and mortality in newborns. It presents a diagnostic challenge to the neonatologists due to a lack of objective evaluation. It may mimic noninfective conditions, such as inborn error of metabolism, birth asphyxia, and even respiratory distress syndrome in preterms. Nonetheless, over-diagnosis and initiating unwanted empirical antibiotics may pose the threat of drug resistance, increasing the hospital stay and cost of treatment. Traditionally, investigations such as white blood cell count, absolute neutrophil count, immature to total neutrophil ratio, C-reactive protein levels, and blood cultures have been used to diagnose sepsis. However, these have low sensitivity and specificity because they may be elevated in conditions other than sepsis. The in-depth understanding of the neonatal immune system’s response to early infection has led to the discovery of advanced diagnostic tools, including biomarkers.\u0000\u0000This literature review briefs on the various haematological parameters and biomarkers in neonatal sepsis, exploring newer biomarkers and comparing them with their older counterparts. This will help early diagnosis, treatment, and improved prognosis in neonatal sepsis. As there is a spectrum of markers for diagnosing neonatal sepsis, it is preferable to compile these markers and correlate clinically.\u0000\u0000A thorough search of this literature was done on the electronic databases PubMed, Elsevier’s Web of Science, and the Cochrane Library. The authors found around 90 relevant articles: 84 were from PubMed, 4 from Elsevier, and 2 from the latest Cochrane database. Of these articles, 57 were selected from between early 2000 and January 2019.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2019-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"130326398","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}
Pub Date : 2019-08-01DOI: 10.33590/emjhematol/10313044
{"title":"Umbilical Cord Blood Donation: An Evolving Lifeline for the Stem Cell Field","authors":"","doi":"10.33590/emjhematol/10313044","DOIUrl":"https://doi.org/10.33590/emjhematol/10313044","url":null,"abstract":"","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2019-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"125178896","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}
Pub Date : 2019-08-01DOI: 10.33590/emjhematol/10311304
E. Zengin, N. Sarper, S. A. Gelen, S. Topçu
Pleural effusion is a common adverse effect of dasatinib, but chylous effusion is rarely reported. Herein, the authors report the case of a 21-year-old imatinib-resistant patient who presented with bilateral massive chylous effusion on Month 44 of dasatinib treatment. The patient was managed with dasatinib withdrawal, bilateral thorax tube insertion, nasal oxygen support, diuretics, corticosteroids, a fat and oil free diet, and sandostatin. The patient required total parenteral nutrition and albumin infusion. The patient’s right lung collapsed as a result of pleural thickening. A subsequent switch to nilotinib was well tolerated. The authors highlight that patients on dasatinib treatment must be carefully followed for adverse effects.
{"title":"Resistant Bilateral Chylous Effusion as a Late Adverse Effect of Dasatinib Treatment in Chronic Myeloid Leukaemia","authors":"E. Zengin, N. Sarper, S. A. Gelen, S. Topçu","doi":"10.33590/emjhematol/10311304","DOIUrl":"https://doi.org/10.33590/emjhematol/10311304","url":null,"abstract":"Pleural effusion is a common adverse effect of dasatinib, but chylous effusion is rarely reported. Herein, the authors report the case of a 21-year-old imatinib-resistant patient who presented with bilateral massive chylous effusion on Month 44 of dasatinib treatment. The patient was managed with dasatinib withdrawal, bilateral thorax tube insertion, nasal oxygen support, diuretics, corticosteroids, a fat and oil free diet, and sandostatin. The patient required total parenteral nutrition and albumin infusion. The patient’s right lung collapsed as a result of pleural thickening. A subsequent switch to nilotinib was well tolerated. The authors highlight that patients on dasatinib treatment must be carefully followed for adverse effects.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2019-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"128573839","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}
Pub Date : 2019-04-08DOI: 10.33590/emjhematol/10314634
J. Fricker
Prof Salles provided an update on the ongoing first-line follicular lymphoma (FL) studies, demonstrating how analysis of the GALLIUM study data regarding the use of different chemotherapy backbones consistently showed the benefits of obinutuzumab (G) chemotherapy (G-chemo) versus rituximab (R) chemotherapy (R-chemo) in FL patients. An update from the PRIMA study showed that 10-year progression-free survival (PFS) was improved following the use of R maintenance compared with observation following induction. Prof Salles also provided an overview of the RELEVANCE study data, which showed that R plus lenalidomide was not superior to standard R-chemo for the treatment of first-line FL. Prof Seymour presented data showing that two-thirds of premature FL deaths occur in patients experiencing disease progression within 2 years of treatment, highlighting the need to identify patients at early risk of progression. Prof Seymour explored various prognostic and predictive tools that could be used to identify patients at high risk of death, but he noted that until these prognostic tools are available in the clinic, the high-risk population remains unidentifiable. Furthermore, the accuracy of these prognostic indices needs to be improved. Data analysis from the GALLIUM study showed that G-chemo decreased the risk of a disease progression event in the first 2 years by 46% compared to R-chemo. Prof Trotman explored the use of PET imaging and detection of minimal residual disease (MRD) to assess treatment outcomes. Prof Trotman showed that PET status at the end of induction is highly prognostic of the outcome. An exploratory analysis of the GALLIUM data showed that the application of the Lugano 2014 response criteria showed a rapid, deep separation of the PFS curves of patients achieving a complete metabolic response (CMR) versus those who did not. There was almost a 5-fold increase in risk of progression and in risk of death in patients failing to achieve CMR. An exploratory analysis of the MRD status of GALLIUM patients showed that a greater proportion of patients in the G-chemo arm achieved MRD-negative status at the end of induction (EOI). Interestingly, patients achieved similar MRD outcomes with G-chemo, regardless of chemotherapy backbone. Both PET and MRD status after induction were independently predictive of PFS. Dr Pettengell and Mr Bouguet discussed FL from both the clinician’s and the patient’s perspective, with a focus on health-related quality of life (HRQoL). They demonstrated how a marked decrease in HRQoL at progression highlights the importance of extending remission for FL patients. Dr Pettengell presented data from GALLIUM showing comparable quality of life for patients treated with either G-chemo or R-chemo. They also presented patient surveys showing that FL has a substantial physical and psychological impact on patients that both lasts beyond treatment and persists even during long-term remission.
{"title":"Expectations and Experience: Optimising Efficacy and Maintaining Quality of Life in Follicular Lymphoma","authors":"J. Fricker","doi":"10.33590/emjhematol/10314634","DOIUrl":"https://doi.org/10.33590/emjhematol/10314634","url":null,"abstract":"Prof Salles provided an update on the ongoing first-line follicular lymphoma (FL) studies, demonstrating how analysis of the GALLIUM study data regarding the use of different chemotherapy backbones consistently showed the benefits of obinutuzumab (G) chemotherapy (G-chemo) versus rituximab (R) chemotherapy (R-chemo) in FL patients. An update from the PRIMA study showed that 10-year progression-free survival (PFS) was improved following the use of R maintenance compared with observation following induction. Prof Salles also provided an overview of the RELEVANCE study data, which showed that R plus lenalidomide was not superior to standard R-chemo for the treatment of first-line FL.\u0000\u0000Prof Seymour presented data showing that two-thirds of premature FL deaths occur in patients experiencing disease progression within 2 years of treatment, highlighting the need to identify patients at early risk of progression. Prof Seymour explored various prognostic and predictive tools that could be used to identify patients at high risk of death, but he noted that until these prognostic tools are available in the clinic, the high-risk population remains unidentifiable. Furthermore, the accuracy of these prognostic indices needs to be improved. Data analysis from the GALLIUM study showed that G-chemo decreased the risk of a disease progression event in the first 2 years by 46% compared to R-chemo.\u0000\u0000Prof Trotman explored the use of PET imaging and detection of minimal residual disease (MRD) to assess treatment outcomes. Prof Trotman showed that PET status at the end of induction is highly prognostic of the outcome. An exploratory analysis of the GALLIUM data showed that the application of the Lugano 2014 response criteria showed a rapid, deep separation of the PFS curves of patients achieving a complete metabolic response (CMR) versus those who did not. There was almost a 5-fold increase in risk of progression and in risk of death in patients failing to achieve CMR. An exploratory analysis of the MRD status of GALLIUM patients showed that a greater proportion of patients in the G-chemo arm achieved MRD-negative status at the end of induction (EOI). Interestingly, patients achieved similar MRD outcomes with G-chemo, regardless of chemotherapy backbone. Both PET and MRD status after induction were independently predictive of PFS.\u0000\u0000Dr Pettengell and Mr Bouguet discussed FL from both the clinician’s and the patient’s perspective, with a focus on health-related quality of life (HRQoL). They demonstrated how a marked decrease in HRQoL at progression highlights the importance of extending remission for FL patients. Dr Pettengell presented data from GALLIUM showing comparable quality of life for patients treated with either G-chemo or R-chemo. They also presented patient surveys showing that FL has a substantial physical and psychological impact on patients that both lasts beyond treatment and persists even during long-term remission.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2019-04-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"130235887","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}
Pub Date : 2018-08-02DOI: 10.33590/emjhematol/10312666
Xinjian Chen
The humanised anti-CD20 antibody (Ab) rituximab (RTX) has significantly improved the prognosis of B cell non-Hodgkin’s lymphomas (BNHL). However, major challenges remain: a) RTX is often used with toxic chemotherapy that not only causes serious side effects but may also compromise RTX activity and host antitumour immunity, predisposing patients to relapse; b) indolent low-grade BNHL remain largely incurable; c) a significant percentage of aggressive BNHL do not respond to RTX-based therapy; and d) a significant number of responders may eventually relapse in long-term follow-up. The data suggest that the limit in the efficacy may result from the inability of RTX to directly kill lymphoma cells. RTX primarily relies on indirect mechanisms to attack lymphoma cells, which include complement-dependent cytotoxicity, Ab-dependent cellular cytotoxicity, induction of apoptosis, and immune activation. These mechanisms could be readily compromised by various situations, such as chemotherapy. The new generation of anti-CD20 Ab have not been found to be directly cytotoxic. Cytotoxic radioactive isotope-conjugated anti-CD20 Ab appeared to be highly effective, but serious radiotoxicity prohibited their clinical application. Increasing Ab valency augments activity; a recent study has demonstrated drastic improvement in activity by non-covalently associating RTX with nanomaterial graphene oxide (GO). The multivalent Ab product RTX/GO is highly cytotoxic, capable of directly killing BNHL cells in vitro and rapidly eliminating established xenograft lymphoma in vivo in the absence of toxic chemo-agents. While further studies are needed to determine the mechanism of activity and clinical efficacy, the current data suggest a significant possibility that RTX/GO might constitute nontoxic but effective therapy for BNHL.
{"title":"Making Rituximab Directly Cytotoxic for Substantial Improvement in Therapeutic Efficacy","authors":"Xinjian Chen","doi":"10.33590/emjhematol/10312666","DOIUrl":"https://doi.org/10.33590/emjhematol/10312666","url":null,"abstract":"The humanised anti-CD20 antibody (Ab) rituximab (RTX) has significantly improved the prognosis of B cell non-Hodgkin’s lymphomas (BNHL). However, major challenges remain: a) RTX is often used with toxic chemotherapy that not only causes serious side effects but may also compromise RTX activity and host antitumour immunity, predisposing patients to relapse; b) indolent low-grade BNHL remain largely incurable; c) a significant percentage of aggressive BNHL do not respond to RTX-based therapy; and d) a significant number of responders may eventually relapse in long-term follow-up. The data suggest that the limit in the efficacy may result from the inability of RTX to directly kill lymphoma cells. RTX primarily relies on indirect mechanisms to attack lymphoma cells, which include complement-dependent cytotoxicity, Ab-dependent cellular cytotoxicity, induction of apoptosis, and immune activation. These mechanisms could be readily compromised by various situations, such as chemotherapy. The new generation of anti-CD20 Ab have not been found to be directly cytotoxic. Cytotoxic radioactive isotope-conjugated anti-CD20 Ab appeared to be highly effective, but serious radiotoxicity prohibited their clinical application. Increasing Ab valency augments activity; a recent study has demonstrated drastic improvement in activity by non-covalently associating RTX with nanomaterial graphene oxide (GO). The multivalent Ab product RTX/GO is highly cytotoxic, capable of directly killing BNHL cells in vitro and rapidly eliminating established xenograft lymphoma in vivo in the absence of toxic chemo-agents. While further studies are needed to determine the mechanism of activity and clinical efficacy, the current data suggest a significant possibility that RTX/GO might constitute nontoxic but effective therapy for BNHL.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2018-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"124993843","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}
Pub Date : 2018-08-02DOI: 10.33590/emjhematol/10310896
Nina Kim, S. Navada
Although hypomethylating agents (HMA) have revolutionised the treatment of myelodysplastic syndromes (MDS), a significant proportion of patients either fail to respond to HMA or their disease progresses after an initial response. Established therapeutic options for these patients remain limited. Fortunately, recent advancements in the knowledge of MDS pathogenesis have allowed for the development of many targeted therapies, including epigenetic regulators, signal transduction regulators, immune checkpoint inhibitors, cell apoptosis regulators, and novel cytotoxic agents. These novel therapeutics have shown varying degrees of promise in clinical trials. Epigenetic regulators, such as second-generation HMA and isocitrate dehydrogenase inhibitors, have shown modest efficacy in early studies, while histone deacetylase inhibitors have, thus far, failed to show significant clinical benefit. Signal transduction modulators, such as transforming growth factor (TGF)-β inhibitors and toll-like receptor inhibitors, appear to alleviate anaemia symptoms, but further studies are needed to determine their effect on survival. Rigosertib, a multikinase inhibitor, improved survival in a small subset of patients with very high-risk MDS. Immune checkpoint inhibitors have shown mixed results. Agents that have recently been approved for use in specific types of high-risk acute myeloid leukaemia, including FMS-like tyrosine receptor kinase 3 inhibitors and CPX-351, are also being studied for use in MDS, with early studies suggesting efficacy. Several other agents are also under investigation with results pending. These novel agents represent potential therapeutic options for patients who have failed HMA and for whom no currently established therapies are available.
{"title":"Therapeutic Options in Myelodysplastic Syndromes: Established and Emerging Therapies","authors":"Nina Kim, S. Navada","doi":"10.33590/emjhematol/10310896","DOIUrl":"https://doi.org/10.33590/emjhematol/10310896","url":null,"abstract":"Although hypomethylating agents (HMA) have revolutionised the treatment of myelodysplastic syndromes (MDS), a significant proportion of patients either fail to respond to HMA or their disease progresses after an initial response. Established therapeutic options for these patients remain limited. Fortunately, recent advancements in the knowledge of MDS pathogenesis have allowed for the development of many targeted therapies, including epigenetic regulators, signal transduction regulators, immune checkpoint inhibitors, cell apoptosis regulators, and novel cytotoxic agents. These novel therapeutics have shown varying degrees of promise in clinical trials. Epigenetic regulators, such as second-generation HMA and isocitrate dehydrogenase inhibitors, have shown modest efficacy in early studies, while histone deacetylase inhibitors have, thus far, failed to show significant clinical benefit. Signal transduction modulators, such as transforming growth factor (TGF)-β inhibitors and toll-like receptor inhibitors, appear to alleviate anaemia symptoms, but further studies are needed to determine their effect on survival. Rigosertib, a multikinase inhibitor, improved survival in a small subset of patients with very high-risk MDS. Immune checkpoint inhibitors have shown mixed results. Agents that have recently been approved for use in specific types of high-risk acute myeloid leukaemia, including FMS-like tyrosine receptor kinase 3 inhibitors and CPX-351, are also being studied for use in MDS, with early studies suggesting efficacy. Several other agents are also under investigation with results pending. These novel agents represent potential therapeutic options for patients who have failed HMA and for whom no currently established therapies are available.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2018-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"114901187","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}
Pub Date : 2018-08-02DOI: 10.33590/emjhematol/10312853
Ahmet Emre Emre Eskazan, M. Tiribelli
The introduction of tyrosine kinase inhibitors (TKI) has dramatically improved the prognosis of chronic myeloid leukaemia (CML) patients and, therefore, changed the therapeutic scenario of this disease. Before the advent of the first TKI imatinib, allogeneic haematopoietic stem cell transplantation (alloSCT) was the only curative approach for CML, and all patients deemed eligible for transplant were referred to a centre for transplant where possible. Nowadays, with the wide availability of five different TKI, indications to alloSCT have been reduced to only include patients in the advanced phase of CML and those with multiple TKI treatment failures. Nonetheless, even in the TKI era, alloSCT retains its curative potential. Herein, the authors give an overview of the indications to allogeneic transplant for CML and the management of TKI in the pre and post-transplant settings.
{"title":"Allogeneic Haematopoietic Stem Cell Transplantation for Chronic Myeloid Leukaemia in the Era of Tyrosine Kinase Inhibitors","authors":"Ahmet Emre Emre Eskazan, M. Tiribelli","doi":"10.33590/emjhematol/10312853","DOIUrl":"https://doi.org/10.33590/emjhematol/10312853","url":null,"abstract":"The introduction of tyrosine kinase inhibitors (TKI) has dramatically improved the prognosis of chronic myeloid leukaemia (CML) patients and, therefore, changed the therapeutic scenario of this disease. Before the advent of the first TKI imatinib, allogeneic haematopoietic stem cell transplantation (alloSCT) was the only curative approach for CML, and all patients deemed eligible for transplant were referred to a centre for transplant where possible. Nowadays, with the wide availability of five different TKI, indications to alloSCT have been reduced to only include patients in the advanced phase of CML and those with multiple TKI treatment failures. Nonetheless, even in the TKI era, alloSCT retains its curative potential. Herein, the authors give an overview of the indications to allogeneic transplant for CML and the management of TKI in the pre and post-transplant settings.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2018-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"133569329","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}
Pub Date : 2018-08-02DOI: 10.33590/emjhematol/10313377
J. Fricker
The meeting was arranged as a series of conversations between experts, following a question and answer format with two speakers in each presentation. In the first presentation, Dr Soverini and Prof Lion discussed the importance of the timing and depth of response with respect to clinical outcomes in Philadelphia chromosome positive (Ph+) leukaemias. They showed how sensitive and reproducible measurements of molecular response (MR) and the proper interpretation of laboratory data are critical to correctly inform therapeutic decisions in patients with chronic myeloid leukaemia (CML) and Ph+ acute lymphoblastic leukaemias (ALL). Detection of BCR-ABL mutations can establish the need for treatment change and, in some cases, indicate which tyrosine-kinase inhibitor (TKI) is most likely to be effective. The speakers addressed the need for more sensitive and accurate methods to monitor minimal residual disease (MRD) and detect mutations that drive resistance to TKI therapy. They explored two distinct patterns of mutation observed in patients with >1 mutation (polyclonal and compound mutations) and how in addition to selecting the most appropriate TKI it is also important to consider the most appropriate dose. In the second presentation, Dr Bassan and Prof Dr Junghanß discussed the evolving treatment landscape for Ph+ ALL, including the role of TKI, chemotherapy, and allogenic stem cell transplantation (SCT). The advent of TKI has improved the prognosis for Ph+ ALL, allowing many more patients to achieve complete remission and be considered for allogeneic SCT. However, treatment-related mortality remains a significant issue after allogenic SCT affecting 20–33% of patients. Studies show that early death rates are lower for patients receiving ‘light’ chemotherapy and TKI with steroids in place of chemotherapy. Furthermore, for patients achieving complete MR, in some studies there is no difference in outcome between those who undergo allogenic SCT and those who do not, provided that the latter subgroup was selected according to absence of residual disease by PCR analysis. Such data suggest that, in Ph+ ALL, novel therapeutic approaches may in some patients obviate the need for intensive chemotherapy and allogeneic SCT. Studies are now ongoing to explore whether Ph+ ALL patients can abstain from allogenic SCT through selection of the strongest TKI upfront and whether chemotherapy-free regimens might be an option.
{"title":"Planning Your Next Move in Philadelphia Chromosome Positive Leukaemias","authors":"J. Fricker","doi":"10.33590/emjhematol/10313377","DOIUrl":"https://doi.org/10.33590/emjhematol/10313377","url":null,"abstract":"The meeting was arranged as a series of conversations between experts, following a question and answer format with two speakers in each presentation. In the first presentation, Dr Soverini and Prof Lion discussed the importance of the timing and depth of response with respect to clinical outcomes in Philadelphia chromosome positive (Ph+) leukaemias. They showed how sensitive and reproducible measurements of molecular response (MR) and the proper interpretation of laboratory data are critical to correctly inform therapeutic decisions in patients with chronic myeloid leukaemia (CML) and Ph+ acute lymphoblastic leukaemias (ALL). Detection of BCR-ABL mutations can establish the need for treatment change and, in some cases, indicate which tyrosine-kinase inhibitor (TKI) is most likely to be effective. The speakers addressed the need for more sensitive and accurate methods to monitor minimal residual disease (MRD) and detect mutations that drive resistance to TKI therapy. They explored two distinct patterns of mutation observed in patients with >1 mutation (polyclonal and compound mutations) and how in addition to selecting the most appropriate TKI it is also important to consider the most appropriate dose.\u0000\u0000In the second presentation, Dr Bassan and Prof Dr Junghanß discussed the evolving treatment landscape for Ph+ ALL, including the role of TKI, chemotherapy, and allogenic stem cell transplantation (SCT). The advent of TKI has improved the prognosis for Ph+ ALL, allowing many more patients to achieve complete remission and be considered for allogeneic SCT. However, treatment-related mortality remains a significant issue after allogenic SCT affecting 20–33% of patients.\u0000\u0000Studies show that early death rates are lower for patients receiving ‘light’ chemotherapy and TKI with steroids in place of chemotherapy. Furthermore, for patients achieving complete MR, in some studies there is no difference in outcome between those who undergo allogenic SCT and those who do not, provided that the latter subgroup was selected according to absence of residual disease by PCR analysis. Such data suggest that, in Ph+ ALL, novel therapeutic approaches may in some patients obviate the need for intensive chemotherapy and allogeneic SCT. Studies are now ongoing to explore whether Ph+ ALL patients can abstain from allogenic SCT through selection of the strongest TKI upfront and whether chemotherapy-free regimens might be an option.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2018-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"128352775","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}
Pub Date : 2018-08-02DOI: 10.33590/emjhematol/10314911
Avantika Gupta, Avanthi Gadipudi
Anaemia is the most widespread of the haematological disorders, affecting about one-third of the global population. Despite decades of public health interventions, anaemia in pregnancy remains a major health problem worldwide, with an estimated 41.8% of pregnant women being diagnosed with anaemia at some point in their gestation. At least half of the cases of anaemia in pregnant women are assumed to be due to iron deficiency, with folate or vitamin B12 deficiency, chronic inflammatory disorders, parasitic infections like malaria, and certain inherited disorders accounting for the remaining cases. A considerable variation has been observed in the incidence and aetiology of iron deficiency anaemia among developed and developing nations, warranting differences in the screening protocols and management strategies used by clinicians in these countries. This article highlights the differences in the management of iron deficiency anaemia among low and high-income countries, with a detailed review of the policies followed in India.
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Pub Date : 2018-08-02DOI: 10.33590/emjhematol/10313097
J. Scarisbrick
Mycosis fungoides (MF) is the most common variant of cutaneous T cell lymphoma and frequently presents as early-stage disease with skin patches and plaques with an indolent course, but patients experience significant morbidity from itch and disfigurement. Around 30% of patients with MF present in the advance stages with skin tumours, erythroderma, and extensive nodal or visceral involvement. Sézary syndrome (SS) is the leukaemic cutaneous T cell lymphoma variant. The staging of MF or SS was revised in 2007 to include skin, nodal, visceral, and blood (tumour- node-metastasis-blood classification) to determine nine stages (IA–IVB). While most patients with early disease (Stages IA–IIA) have a good prognosis, 25% progress to advanced disease, with a poor life expectancy of around 3 years; however, some patients do survive for ≥10 years. Accurate staging is crucial since management strategies are stage-based, with skin-directed therapy recommended in early-stage disease and with no curative therapeutic options to improve symptoms and reduce skin tumour burden. In contrast, advanced-stage patients mostly require systemic therapy. Most treatments have only partial response rates, around 40%, and allogeneic bone marrow transplant may provide a more long-lasting therapeutic option for advanced patients. Relevant prognostic factors within the tumour-node-metastasis-blood classification are discussed in this review and their relevance to overall IA–IVB staging and outcomes are debated. Several important prognostic features have been identified that may be used alongside staging to give further prognostic information. These prognostic features include age >60 years at diagnosis, large cell transformation of the skin, and raised serum lactate dehydrogenase levels, which could be developed into a prognostic index to identify patients at risk of progression and requiring more aggressive therapy. The PROCLIPI study, a prospective cutaneous lymphoma international study, has been ongoing since 2015 to collect such data, with the aim of developing a prognostic index for MF and SS.
{"title":"Staging of Mycosis Fungoides and Sézary Syndrome: Time for an Update?","authors":"J. Scarisbrick","doi":"10.33590/emjhematol/10313097","DOIUrl":"https://doi.org/10.33590/emjhematol/10313097","url":null,"abstract":"Mycosis fungoides (MF) is the most common variant of cutaneous T cell lymphoma and frequently presents as early-stage disease with skin patches and plaques with an indolent course, but patients experience significant morbidity from itch and disfigurement. Around 30% of patients with MF present in the advance stages with skin tumours, erythroderma, and extensive nodal or visceral involvement. Sézary syndrome (SS) is the leukaemic cutaneous T cell lymphoma variant. The staging of MF or SS was revised in 2007 to include skin, nodal, visceral, and blood (tumour- node-metastasis-blood classification) to determine nine stages (IA–IVB). While most patients with early disease (Stages IA–IIA) have a good prognosis, 25% progress to advanced disease, with a poor life expectancy of around 3 years; however, some patients do survive for ≥10 years. Accurate staging is crucial since management strategies are stage-based, with skin-directed therapy recommended in early-stage disease and with no curative therapeutic options to improve symptoms and reduce skin tumour burden. In contrast, advanced-stage patients mostly require systemic therapy. Most treatments have only partial response rates, around 40%, and allogeneic bone marrow transplant may provide a more long-lasting therapeutic option for advanced patients.\u0000\u0000Relevant prognostic factors within the tumour-node-metastasis-blood classification are discussed in this review and their relevance to overall IA–IVB staging and outcomes are debated. Several important prognostic features have been identified that may be used alongside staging to give further prognostic information. These prognostic features include age >60 years at diagnosis, large cell transformation of the skin, and raised serum lactate dehydrogenase levels, which could be developed into a prognostic index to identify patients at risk of progression and requiring more aggressive therapy. The PROCLIPI study, a prospective cutaneous lymphoma international study, has been ongoing since 2015 to collect such data, with the aim of developing a prognostic index for MF and SS.","PeriodicalId":326555,"journal":{"name":"EMJ Hematology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2018-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"132290120","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}
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