Gene therapy for sickle cell disease
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):542-547. doi: 10.1182/hematology.2023000487.ABSTRACTSickle cell disease (SCD) is potentially curable after allogeneic hematopoietic stem cell transplantation (HSCT) or autologous HSCT after ex vivo genetic modification. Autologous HSCT with gene therapy has the potential to overcome many of the limitations of allogeneic HSCT that include the lack of suitable donors, graft-versus-host disease, the need for immune suppression, and the potential for graft rejection. Significant progress in gene therapy for SCD has been made over the past several decades, now with a gr...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Alexis Leonard John F Tisdale Source Type: research
MRD-directed therapy in CLL: ready for prime time?
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):413-420. doi: 10.1182/hematology.2023000441.ABSTRACTIn recent years, the treatment paradigm for patients with chronic lymphocytic leukemia (CLL) has moved away from chemoimmunotherapy (CIT) toward the use of novel targeted agents. Commercially available drugs, including Bruton's tyrosine kinase inhibitors and the BCL2 inhibitor venetoclax, often used in combination with anti-CD20 monoclonal antibodies, are now the mainstay of therapy both in the frontline and in relapsed settings. As the landscape for CLL management evolves, therapeutic endpoints need to be redefin...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Joanna M Rhodes Carlos A Lopez Jacqueline C Barrientos Source Type: research
Amyloid consults do not have to be vexing
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):407-412. doi: 10.1182/hematology.2023000440.ABSTRACTDiagnosing amyloidosis can be challenging due to its clinical heterogeneity, need for multiple specialists to make a diagnosis, and lack of a single diagnostic test for the disease. Patients are often diagnosed late, in advanced stage, and after exhibiting multiple symptoms and signs for a long period. It is important to develop a clinical suspicion of amyloidosis, particularly in those with multisystemic symptoms and high-risk patient populations such as those with precursor hematologic conditions. A systematic a...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Anita D'Souza Source Type: research
IV iron formulations and use in adults
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):622-629. doi: 10.1182/hematology.2023000495.ABSTRACTIntravenous iron has become a major component of the therapeutic armamentarium for iron deficiency and iron deficiency anemia. The earliest formulations were associated with unacceptable toxicity. Newer formulations, with complex carbohydrate cores that bind elemental iron more tightly, allow the administration of full therapeutic doses in 15 to 60 minutes. Nonetheless, a folklore of danger, fueled by earlier formulations no longer available, continues to foment caution. Complement-mediated minor infusion reaction...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Layla Van Doren Michael Auerbach Source Type: research
Sex, lies, and iron deficiency: a call to change ferritin reference ranges
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):617-621. doi: 10.1182/hematology.2023000494.ABSTRACTIron deficiency is a very common and treatable disorder. Of all the tests available to diagnose iron deficiency, the serum ferritin is the most able to discriminate iron deficiency from other disorders. However, the reference range for ferritin in many laboratories will lead to underdiagnosis of iron deficiency in women. Studies have shown that 30%-50% of healthy women will have no marrow iron stores, so basing ferritin cutoffs on the lowest 2.5% of sampled ferritins is not appropriate. In addition, several lines ...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Kylee Martens Thomas G DeLoughery Source Type: research
Mutational screening to improve the transplantation decision-making process in MDS
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):73-76. doi: 10.1182/hematology.2023000516.NO ABSTRACTPMID:38066932 | PMC:PMC10727083 | DOI:10.1182/hematology.2023000516 (Source: Hematology ASH Education Program)
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Alessia Campagna Matteo G Della Porta Source Type: research
Labor and delivery: DIC, HELLP, preeclampsia
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):737-744. doi: 10.1182/hematology.2023000500.ABSTRACTHematologists are often needed to assist with the management of microangiopathic emergencies in pregnancy. A firm understanding of the diagnosis and management of preeclampsia with severe features, hemolysis elevated liver enzyme and low platelet syndrome, and disseminated intravascular coagulation, which are the most common causes of microangiopathic emergencies, is critical. However, being able to consider when other microangiopathic emergencies (acute fatty liver of pregnancy, congenital and acquired thrombotic...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Juliana Perez Botero Jennifer Jury McIntosh Source Type: research
What makes a pediatric or young adult patient an appropriate transplant candidate?
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):731-736. doi: 10.1182/hematology.2023000519.ABSTRACTA 3-year-old child with chronic granulomatous disease was brought to the transplant clinic by his parents. The patient has a history of Aspergillus fumigatus pneumonia, which required mechanical ventilation, and sepsis, resulting in several intensive care stays. He has failure to thrive and developmental delay. His parents are seeking guidance whether allogeneic hematopoietic cell transplantation (HCT) is a reasonable treatment option given concerns about his upfront major health limitations. Based on the original...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Monica S Thakar Mohamed L Sorror Source Type: research
Approaches to optimize outcomes in transplant recipients
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):723-730. doi: 10.1182/hematology.2023000459.ABSTRACTCurative therapy with an allogeneic hematopoietic cell transplant (HCT) can now be offered to a wider patient population due to improvements in donor selection, transplant conditioning regimens, and supportive care measures. However, risk of transplant-related morbidity and mortality remains, and thus appropriate transplant candidate workup pre-HCT for risk stratification and a management plan after HCT is crucial for success of the procedure. These include understanding and identifying risk of underlying malignan...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Asmita Mishra Source Type: research
Provoked vs minimally provoked vs unprovoked VTE: does it matter?
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):600-605. doi: 10.1182/hematology.2023000492.ABSTRACTVenous thromboembolism (VTE) is a multifactorial disease, and its risk depends on exposure to risk factors and predisposing conditions. Based on their strength of association with a VTE episode, risk factors are classified as major or minor and determined using a temporal pattern to be transient or persistent. All patients with VTE should receive anticoagulant treatment for at least 3 months in the absence of an absolute contraindication. Beyond this period, selected patients may be candidates for an extended phas...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Cecilia Becattini Ludovica Anna Cimini Source Type: research
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
