Publications by authors named "N Varda-Bloom"
Article Synopsis
- Pearson syndrome (PS) and Kearns-Sayre syndrome (KSS) are mitochondrial DNA deletion syndromes with PS causing severe childhood cytopenia and KSS having later onset without blood-related issues, both sharing a common mitochondrial DNA deletion.
- A study of 16 patients revealed that 75% had cytopenia, with many needing blood transfusions, and even after achieving transfusion independence, they showed persistent bone marrow (BM) dysfunction.
- The research highlights that BM dysfunction is a consistent finding in SLSMD syndromes, which raises concerns about potential clonal evolution and chromosome 7 abnormalities, underscoring the need for specialized hematological monitoring for these patients.
Background: Haploidentical hematopoietic stem cell transplantation (HSCT) with depletion of αβ T cells and CD19 B cells has emerged as a viable alternative to traditional donors for treating acute leukemia in children. As the use of this innovative approach continues to grow and more experience is gained, it is essential to identify and comprehend the key factors that contribute to successful transplantation and improved outcomes.
Methods: We conducted a retrospective analysis of single-center data from 27 children with acute lymphoblastic leukemia and 11 children with acute myeloid leukemia who underwent haploidentical HSCT with depletion of αβ T cells and CD19 B cells between the years 2013 and 2020.
Article Synopsis
- Patients with single large-scale mitochondrial DNA (mtDNA) deletion syndromes (SLSMDs) often suffer from serious multisystemic diseases like Pearson syndrome in childhood or Kearns-Sayre syndrome later on, and there's currently no effective treatment available.
- A new approach called mitochondrial augmentation therapy (MAT) was tested on six patients with SLSMDs, where their own hematopoietic cells were infused with healthy maternal mitochondria.
- The treatment showed safety, decreased harmful mtDNA levels in four patients, and increased mtDNA content in all six, alongside some improvements in aerobic function and quality of life measured by caregivers, suggesting a need for further clinical trials.
Tisagenlecleucel (tisa-cel) is an anti-CD19 chimeric antigen receptor (CAR) T-cell therapy approved for patients with relapsed/refractory large B-cell lymphoma. Outcomes of patients with out-of-commercial specification (OOS) CAR T products are not well characterized. We therefore assessed 37 adult patients who underwent leukapheresis for tisa-cel therapy in a single center.
View Article and Find Full Text PDFAutologous stem cell transplantation (ASCT) is a standard of care in newly-diagnosed multiple myeloma (MM) patients. Several studies before the introduction of novel therapies in MM, demonstrated a pegylated G-CSF to be successful in mobilizing peripheral blood stem cells (PBSCs). Lipegfilgrastim is a novel long-acting G-CSF that is produced by the conjugation of a single 20-kDa polyethelene glycol to the natural O-glycosylation site of G-CSF.
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