Publications by authors named "B Bielorai"
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.
Article Synopsis
- Inotuzumab ozogamicin is an approved treatment for relapsed/refractory B-cell precursor acute lymphoblastic leukemia (BCP-ALL) in adults, but its pharmacokinetics in children have not been studied until now.
- This research analyzed serum concentrations from a diverse patient group, utilizing a modified adult pharmacokinetic model to understand how the drug behaves in pediatric patients with BCP-ALL.
- Results indicated that older pediatric patients showed a different drug clearance pattern, with higher drug exposure correlating to better treatment responses among those with relapsed/refractory BCP-ALL.
Article Synopsis
- Congenital neutropenias in Israel show a diverse genetic landscape, with a focus on severe cases, particularly highlighting the prevalence of G6PC3 variants in consanguineous populations.
- A total of 65 patients were analyzed, revealing that the majority of severe cases were linked to ELANE and G6PC3 mutations, while some had other genetic variants or conditions like cyclic neutropenia or Shwachman-Diamond syndrome.
- The study emphasizes the importance of ongoing monitoring for patients due to a significant occurrence of myeloid transformation, indicating gaps in medical understanding as some patients remain without a clear genetic diagnosis.
Inotuzumab ozogamicin (InO) is a CD22-directed antibody conjugated with calicheamicin. The phase IB of the ITCC-059 trial tested InO combined with chemotherapy in pediatric B-cell precursor acute lymphoblastic leukemia (BCP-ALL). Relapsed /refractory CD22+ BCP-ALL pediatric patients were enrolled.
View Article and Find Full Text PDFThe presence of leukocytes in the cerebral spinal fluid (CSF) of patients with acute lymphoblastic leukemia may indicate a relapse in the central nervous system. CD19-directed immunotherapy may increase the blood-brain barrier permeability, leading to neurologic toxicity and infiltrate the CNS. We studied the CSF cell and protein content in 71 consecutive patients who received either CD19 chimeric antigen receptor T cells or blinatumomab.
View Article and Find Full Text PDF