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
