A study assessing the feasibility of randomization of pediatric and young adult patients between matched unrelated donor bone marrow transplantation and immune-suppressive therapy for newly diagnosed severe aplastic anemia: A joint pilot trial of the North American Pediatric Aplastic Anemia Consortium and the Pediatric Transplantation and Cellular Therapy Consortium.
Michael A PulsipherLeslie E LehmannAlison A BertuchGhadir SasaTimothy OlsonTaizo A NakanoAlfred GilioLauri M BurroughsJeffrey M LiptonJames N HuangKathryn DickersonAlice BertainaCindy ZhuangMaggie MalschMark FlemingEdie WellerAkiko ShimamuraDavid A WilliamsPublished in: Pediatric blood & cancer (2020)
This feasibility study showed that a high percentage of patients underwent randomization and received up-front MUD BMT. Our study lays the groundwork for a larger randomized trial that will define best initial therapy for young patients with SAA who have an available MUD.
Keyphrases
- newly diagnosed
- chronic kidney disease
- end stage renal disease
- bone marrow
- ejection fraction
- allogeneic hematopoietic stem cell transplantation
- mesenchymal stem cells
- cell therapy
- prognostic factors
- middle aged
- iron deficiency
- peritoneal dialysis
- stem cells
- early onset
- patient reported outcomes
- young adults
- childhood cancer