Cyclophosphamide (CTX, Cytoxan)

DRUG2 trials

Sponsors

National Cancer Institute (NCI)

Conditions

GATA2ImmunodeficiencyMDS

Phase 2

Pilot and Feasibility Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for MonoMAC

CompletedNCT00923364

National Cancer Institute (NCI)GATA2, Immunodeficiency, MDS

Start: 2009-05-07End: 2017-02-26Updated: 2020-03-12

Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations

RecruitingNCT01861106

National Cancer Institute (NCI)GATA2, Immunodeficiency, MDS

Start: 2013-07-24End: 2028-12-31Target: 144Updated: 2026-03-24

Related Papers

A novel <i>GATA2</i> distal enhancer mutation results in MonoMAC syndrome in 2 second cousins

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Allogeneic Hematopoietic Stem-Cell Transplantation in Patients with GATA 2 Deficiency: Influence of Donor Stem Cell Source and Post-Transplantation Cyclophosphamide

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Sequencing of RNA in single cells reveals a distinct transcriptome signature of hematopoiesis in GATA2 deficiency

Blood Advances2020-06-1835 citations

Acquired ASXL1 mutations are common in patients with inherited GATA2 mutations and correlate with myeloid transformation

Haematologica2013-09-27135 citations

Successful allogeneic hematopoietic stem cell transplantation for GATA2 deficiency.

2011-08-03103 citations

Myelodysplasia in autosomal dominant and sporadic monocytopenia immunodeficiency syndrome: diagnostic features and clinical implications.

2011-04-2083 citations