Non-Malignant Diseases Working Committee Studies

The Non-Malignant Diseases Working Committee provides scientific oversight for studies related to hematopoietic cell transplantation and gene therapy/gene editing for immune deficiencies, inborn errors of metabolism, autoimmune diseases, aplastic anemia, hemoglobinopathy, and other non-malignant hematopoietic disorders.

Learn more about the studies conducted through the Non-Malignant Diseases Working Committee using the links below.

Recently Completed Studies

Number	Title	Status
NM14-02	Outcomes of allogeneic hematopoietic cell transplant in patients with schwachman diamond syndrome.	Published
CV20-02a	Fresh vs cryo-preserved graft with post-transplant CY in non-malignant diseases.	Published
NM19-03	Hematopoietic stem cell transplantation for congenital amegakaryocytic thrombocytopenia.	Published
NM19-02	Impact of Conditioning Regimen on Allogeneic HCT Outcomes for Hyper-inflammatory Immune Deficiency Disorders	Published
NM17-03b	Risk factor scoring for allogeneic hematopoietic stem cell transplantation for sickle cell disease.	Published

Preliminary Results

Number	Title	Status
NM16-03	Results of transplants from genetically-identical twin donors in persons with aplastic anemia.	Submitted
NM19-01	Conditional and cause-specific mortality of patients with severe aplastic anemia surviving at least one year after allohematopoietic cell transplantation or immunosuppressive therapy.	Manuscript Preparation
AC18-02	Prospective cohort study of recipients of autologous hematopoietic cell transplant for systemic sclerosis.	Manuscript Preparation
NM15-01	Outcome of allogeneic hematopoietic cell transplant in erythropoietic porphyria.	Manuscript Preparation

Studies in Progress

Number	Title	Status
NM18-01	Impact of choice of serotherapy in pediatric stem cell transplantation for non-malignant diseases.	Data File Preparation
NM17-01	Late effects after hematopoietic stem cell transplantation in patients with hemophagocytic lymph histiocytosis.	Data File Preparation
SC17-09 17-CMS-SCD	Prospective assessment of allogeneic hematopoietic cell transplantation in adolescents and young adults with severe sickle cell disease.	Data Collection/Data File Preparation
NM20-01	Hematopoietic stem cell transplantation for fanconi anemia.	Protocol Development

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Non-Malignant Diseases Working Committee Studies

Recently Completed Studies

Preliminary Results

Studies in Progress

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