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Successful treatment with daratumumab for post-HSCT refractory hemolytic anemia.
Pediatr Blood Cancer
January 2020
Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
Autoimmune cytopenias (AIC) following allogeneic hematopoietic stem cell transplantation (HSCT) may cause significant morbidity and mortality and are often challenging to treat. We present a case of a pediatric patient with primary myelofibrosis of infancy caused by VPS45 protein deficiency, who developed severe refractory hemolytic anemia and immune-mediated thrombocytopenia 3.5 months following HSCT.
View Article and Find Full Text PDFA novel homozygous VPS45 p.P468L mutation leading to severe congenital neutropenia with myelofibrosis.
Pediatr Blood Cancer
September 2017
Jimmy Everest Section of Pediatric Hematology-Oncology and Bone Marrow Transplant, Oklahoma University Health Science Center, Oklahoma City, OK.
VPS45-associated severe congenital neutropenia (SCN) is a rare disorder characterized by life-threating infections, neutropenia, neutrophil and platelet dysfunction, poor response to filgrastim, and myelofibrosis with extramedullary hematopoiesis. We present a patient with SCN due to a homozygous c.1403C>T (p.
View Article and Find Full Text PDFVPS 45-associated primary infantile myelofibrosis--successful treatment with hematopoietic stem cell transplantation.
Pediatr Transplant
December 2013
Department of Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Hospital, Jerusalem, Israel.
PMF of infancy is a recently described autosomal recessive disorder presenting with severe bone marrow failure, accelerated neutrophil apoptosis, and significant platelet dysfunction, caused by a mutation in the VPS45 gene. In this study, we update our group of patients with PMF, highlighting different aspects of this disease, and evaluating the effectiveness of HSCT for the treatment of this disorder. Update of clinical data, hematological features, molecular studies, treatment and final outcome of four children diagnosed with VPS 45-associated PMF of infancy.
View Article and Find Full Text PDFAllogeneic hematopoietic stem cell transplantation for infants with idiopathic myelofibrosis.
Pediatr Transplant
December 2013
Bone Marrow and Stem Cell Transplantation Program, King Hussein Cancer Center, Amman, Jordan.
IMF is a rare disease in children that can present during infancy and has a protracted course. The only known curative approach for this disease in adult patients is allogeneic HSCT. There are very few reports describing the long-term outcome of young children following stem cell transplantation for IMF.
View Article and Find Full Text PDFMyelofibrosis, hepatic extramedullary hematopoiesis and ascites associated with vitamin D deficiency in early infancy.
J Pediatr Hematol Oncol
May 2014
*Carman and Ann Adams Department of Pediatrics, Division of Hematology/Oncology Divisions of ‡Critical Care §Pathology, Children's Hospital of Michigan †Wayne State University, Detroit, MI.
Primary myelofibrosis is rare in children. Many causes have been described for secondary myelofibrosis including vitamin D deficiency. Here, we describe a patient with myelofibrosis secondary to vitamin D deficiency, as diagnosed by laboratory evidence.
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