Secondary Malignancies After Allogeneic Hematopoietic Stem Cell Transplantation for Sickle Cell Disease Inform Gene Therapy Approaches

Article Oncology

Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease

Mary Eapen et al.

Summary: This study investigates the incidence and risk factors for secondary neoplasm after transplantation for sickle cell disease. The results show that the 10-year incidence of leukemia/MDS was 1.7% and of any secondary neoplasm was 2.4%. Low-intensity regimens were associated with higher risks for leukemia/MDS or any secondary neoplasm compared with more intense regimens.

JOURNAL OF CLINICAL ONCOLOGY (2023)

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Article Medicine, General & Internal

Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease

Julie Kanter et al.

Summary: The LentiGlobin gene therapy for sickle cell disease showed promising results, with significant improvements in hemoglobin levels and complete resolution of severe vaso-occlusive events in 35 patients. Adverse events related to treatment were minimal and no cases of hematologic cancer were observed during the follow-up period. Preliminary findings suggest the potential therapeutic efficacy of this treatment.

NEW ENGLAND JOURNAL OF MEDICINE (2022)

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Article Medicine, General & Internal

Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease

Sunita Goyal et al.

Summary: A woman developed acute myeloid leukemia 5.5 years after receiving LentiGlobin therapy for sickle cell disease, but investigation revealed that the leukemia was unlikely related to vector insertion. Other genetic mutations and inadequate disease control after treatment may contribute to the development of leukemia in sickle cell disease patients.

NEW ENGLAND JOURNAL OF MEDICINE (2022)

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Article Hematology

Inflammation and myeloid malignancy: quenching the flame

Ryan J. Stubbins et al.

Summary: Chronic inflammation with aging, known as inflammaging, plays a significant role in the development of myeloid malignancies. Aberrant inflammatory activity affects various cells in the bone marrow, including normal blood and stromal cells, as well as leukemic cells, in distinct ways. Inflammation can selectively promote the clonal expansion of malignant cells by suppressing normal hematopoietic cells through immune-mediated mechanisms. Understanding the intrinsic and extrinsic effects of inflammation provides insights for future clinical trials.

BLOOD (2022)

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Letter Hematology

Increased incidence of hematologic malignancies in SCD after HCT in adults with graft failure and mixed chimerism

Rialnat A. Lawal et al.

BLOOD (2022)

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Article Medicine, General & Internal

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

H. Frangoul et al.

Summary: Research utilizing CRISPR-Cas9 technology for gene editing in patients with TDT and SCD showed high levels of edited alleles in bone marrow and blood, leading to partial or complete elimination of symptoms.

NEW ENGLAND JOURNAL OF MEDICINE (2021)

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Article Hematology