Medical consult: aHUS, TTP? How to distinguish and what to do
We present an evidence-based approach to the initial (first 24 hours) diagnosis and management of iTTP and review the clinical and laboratory features that can be used to identify patients with aHUS who will benefit from early C5 blockade. We also discuss the potential use of complement blockade to improve outcomes in selected patients with secondary TMA.PMID:38066937 | PMC:PMC10727109 | DOI:10.1182/hematology.2023000501 (Source: Hematology ASH Education Program)
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Charlotte M Story Gloria F Gerber Shruti Chaturvedi Source Type: research
Management of limited-stage Hodgkin lymphoma
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):500-509. doi: 10.1182/hematology.2023000511.ABSTRACTHodgkin lymphoma (HL) is a rare type of B-cell malignancy with bimodal age distribution targeting young adults and elderly. Prognostic models are available to identify risk of recurrence and response to treatment. Currently, positron emission tomography scanning is most useful in optimizing therapy. Outcomes are generally excellent with standard chemotherapy or combined modality therapy. Balancing efficacy and the risk of late effects in Hodgkin lymphoma is essential, including early detection of potential complic...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Taha Al-Juhaishi Sairah Ahmed Source Type: research
Consumptive coagulopathy in the ICU
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):754-760. doi: 10.1182/hematology.2023000502.ABSTRACTA consumptive coagulopathy describes a situation where there is a loss of hemostatic factors, which leads to an increased risk of bleeding. Some recent studies have used the term interchangeably with disseminated intravascular coagulation (DIC), but we have reverted to the older definition, which covers a broader range of issues where there is loss of hemostatic factors due to multiple causes, which includes systemic activation of coagulation as seen in DIC. Therefore, the term consumptive coagulopathy covers cond...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Andrew Retter Beverley J Hunt Source Type: research
Pyruvate kinase activators for treatment of pyruvate kinase deficiency
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):97-106. doi: 10.1182/hematology.2023000466.ABSTRACTPyruvate kinase (PK) deficiency is a congenital hemolytic anemia with wide-ranging clinical symptoms and complications associated with significant morbidity and reduced health-related quality of life in both children and adults. The management of patients with PK deficiency has been historically challenging due to difficulties in the diagnostic evaluation, heterogeneity of clinical manifestations, and treatment options limited to supportive care with transfusions and splenectomy. An oral allosteric PK activator, mi...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Rachael F Grace Source Type: research
Preventing relapse after CD19 CAR T-cell therapy for pediatric ALL: the role of transplant and enhanced CAR T cells
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):91-96. doi: 10.1182/hematology.2023000424.ABSTRACTCD19-specific chimeric antigen receptor (CAR) T-cell therapy has become an integral part of our treatment armamentarium for pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL). However, despite initial remission rates of greater than 80%, durable remission occurs in only 40% to 50% of patients. In this review we summarize our current knowledge of the role of consolidative hematopoietic cell transplantation in the management of pediatric patients who achieved a minimal residual ...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Aimee C Talleur Swati Naik Stephen Gottschalk Source Type: research
The sum of the parts: what we can and cannot learn from comorbidity scores in allogeneic transplantation
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):715-722. doi: 10.1182/hematology.2023000458.ABSTRACTAllogeneic hematopoietic cell transplantation (alloHCT) requires the comprehensive evaluation of patients across multiple dimensions. Among the factors considered, comorbidities hold great significance in the pretransplant assessment. As many as 40% of alloHCT recipients will have a high burden of comorbidities in contemporary cohorts. To ensure a standardized evaluation, several comorbidity scores have been developed; however, they exhibit variations in properties and performance. This review examines the strengt...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Roni Shouval Joshua A Fein Source Type: research
How old is too old? Frailty and geriatric assessments of older patients undergoing allogeneic HCT
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):709-714. doi: 10.1182/hematology.2023000457.ABSTRACTAllogeneic hematopoietic cell transplantation (HCT) is a curative-intent treatment for many hematologic malignancies but carries a significant risk of morbidity and mortality. An increasing number of older adults are receiving HCT, but current pretransplant evaluations overlook the unique vulnerabilities that older adults face. Oncology-specific geriatric and frailty assessments provide a comprehensive evaluation of older adults, help better weigh the risks of HCT with patients, and guide personalized optimization...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Reena V Jayani Source Type: research
The range of haploidentical transplant protocols in sickle cell disease: all haplos are not created equally
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):532-541. doi: 10.1182/hematology.2023000486.ABSTRACTThe ideal curative therapy for sickle cell disease (SCD) must be applicable across all ages and include individuals with strokes and preexisting heart, lung, and kidney disease. Myeloablative, matched sibling donor hematopoietic stem cell transplant (HCT) for children with SCD has shown excellent outcomes over the past 3 decades but has been restricted due to the limited availability of a human leukocyte antigen-matched sibling donor (10%-15%) and increased treatment-related death in adults with myeloablative cond...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Adetola A Kassim Michael R DeBaun Source Type: research
How to classify risk based on clinical and molecular modeling: integrating molecular markers in the risk assessment of myelodysplastic syndrome
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):51-58. doi: 10.1182/hematology.2023000420.ABSTRACTMyelodysplastic syndrome (MDS), also known as "myelodysplastic neoplasm," is a heterogeneous group of clonal myeloid neoplasms that typically affects older adults. The clinical phenotype, symptoms, and complications relate to the depth of cytopenia and progression to acute myeloid leukemia (AML). The diagnosis of MDS relies on morphologic criteria, such as evidence of dysplasia, disordered maturation, and increasing blast counts, which separate the disease into histologic subtypes with different probabilities for pr...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Rena R Xian Source Type: research
A rational approach to functional high-risk myeloma
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):433-442. doi: 10.1182/hematology.2023000443.ABSTRACTMultiple myeloma is a clinically and biologically highly heterogeneous disease, as the overall survival can vary from more than a decade in patients with standard risk disease treated with intensive chemotherapy to 2-3 years in patients with high-risk features. The current staging systems, which rely on baseline biological risk factors to stratify patients into groups with differing risks of progression or death, are sometimes suboptimal tools for identifying high-risk patients. This is particularly evident when c...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Francesca Gay Giuseppe Bertuglia Roberto Mina Source Type: research
Richter transformation-is there light at the end of this tunnel?
This article reviews recent therapeutic developments, focusing on inhibitors of BTK, BCL2, the PD1-PDL1 axis, and T-cell-activating/engaging therapies. Herein, I discuss the importance of randomized clinical trials in a disease where small single-arm studies dominate; industry engagement, including the role of registrational studies; and the need to integrate prospectively planned correlative biological studies embedded within future clinical trials to help discover which patient benefits most from each class or combination of novel targets.PMID:38066897 | PMC:PMC10727096 | DOI:10.1182/hematology.2023000442 (Source: Hemato...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Toby A Eyre Source Type: research
Managing side effects: guidance for use of immunotherapies in multiple myeloma
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):348-356. doi: 10.1182/hematology.2023000435.ABSTRACTChimeric antigen receptor T-cell therapy and bispecific T-cell recruiting antibodies have transformed the treatment landscape for relapsed/refractory multiple myeloma, with B-cell maturation antigen being the most common target and other targets in clinical development. However, these therapies are associated with unique and severe toxicities, including cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), delayed neurotoxicity, cytopenias, and infection. In addition, imm...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Emily C Liang Surbhi Sidana Source Type: research
How to avoid early mortality in acute promyelocytic leukemia
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):248-253. doi: 10.1182/hematology.2023000477.ABSTRACTAcute promyelocytic leukemia (APL), a phenotypically and genotypically unique subtype of acute myeloid leukemia, has seen unprecedented advances in its management since the introduction of all-trans retinoic acid (ATRA) and arsenic trioxide. However, the phenomenal pharmacologic conversion of this once highly fatal disease to one with a long-term survival exceeding 90% among patients who survive induction remains impaired by the significant incidence of early death (ED) reaching 30% in some real-world studies. The...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Oluwatobi Odetola Martin S Tallman Source Type: research
Minimal intensity conditioning strategies for bone marrow failure: is it time for "preventative" transplants?
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):135-140. doi: 10.1182/hematology.2023000470.ABSTRACTHematopoietic cell transplantation (HCT) can cure blood dyscrasias and reduce the risk of hematologic cancers in patients with inherited bone marrow failure syndromes (IBMFS). However, because of its high mortality rate, HCT is generally reserved until patients with IBMFS manifest life-threatening cytopenias or myeloid malignancy, at which point outcomes are poor. Screening tests that accurately predict transformation and enable timely intervention are lacking. These unknowns and risks limit the use of HCT in pati...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Suneet Agarwal Source Type: research
Stem cell transplantation for ALL: you've always got a donor, why not always use it?
This article reviews critical current evidence and debates the potential of HSCT as a more universal treatment for ALL, reevaluating traditional treatment stratification in light of the constant availability of stem cell donors.PMID:38066901 | PMC:PMC10726989 | DOI:10.1182/hematology.2023000423 (Source: Hematology ASH Education Program)
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: David Shyr Kara L Davis Alice Bertaina Source Type: research
