期刊
BLOOD
卷 135, 期 23, 页码 2094-2105出版社
AMER SOC HEMATOLOGY
DOI: 10.1182/blood.2019002939
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资金
- Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases (NIAID)
- Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)
- Public Health Service, NIH, NIAID [U54AI082973]
- NIH, National Institute of Neurological Disorders and Stroke [U54NS064808]
- National Center for Advancing Translational Sciences [U01TR001263]
- NIH, NIAID [R13AI094943]
- PBMTC Operations Center by the St. Baldrick's Foundation
- NIH, National Heart, Lung and Blood Institute (NHLBI) [U10HL069254]
- NIH, National Cancer Institute [U24CA076518]
- NHLBI
- NIAID
- NIH, NHLBI [U01HL069294]
- Health Resources and Services Administration (HRSA/DHHS) [HHSH250201200016C, HHSH234200637015C]
- Office of Naval Research [N00014-13-1-0039, N0001414-1-0028]
- Dejoria Wiskott-Aldrich Research Fund
- Jeffrey Modell Foundation
- Division of Intramural Research, NIH, NIAID grant [1 ZIA AI001222-02]
- Children's Discovery Institute, St. Louis Children's Hospital
- NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES [ZIAAI001222] Funding Source: NIH RePORTER
Wiskott-Aldrich syndrome (WAS) is an X-linked disease caused by mutations in the WAS gene, leading to thrombocytopenia, eczema, recurrent infections, autoimmune disease, and malignancy. Hematopoietic cell transplantation (HCT) is the primary curative approach, with the goal of correcting the underlying immunodeficiency and thrombocytopenia. HCT outcomes have improved over time, particularly for patients with HLA-matched sibling and unrelated donors. We report the outcomes of 129 patients with WAS who underwent HCT at 29 Primary Immune Deficiency Treatment Consortium centers from 2005 through 2015. Median age at HCT was 1.2 years. Most patients (65%) received myeloablative busulfanbased conditioning. With a median follow-up of 4.5 years, the 5-year overall survival (OS) was 91%. Superior 5-year OS was observed in patients <5 vs >= 5 years of age at the time of HCT (94% vs 66%; overall P = .0008). OS was excellent regardless of donor type, even in cord blood recipients (90%). Conditioning intensity did not affect OS, but was associated with donor T-cell and myeloid engraftment after HCT. Specifically, patients who received fludarabine/melphalan-based reduced-intensity regimens were more likely to have donor myeloid chimerism <50% early after HCT. In addition, higher platelet counts were observed among recipients who achieved full (>95%) vs low-level (5%-49%) donor myeloid engraftment. In summary, HCT outcomes for WAS have improved since 2005, compared with prior reports. HCT at a younger age continues to be associated with superior outcomes supporting the recommendation for early HCT. High-level donor myeloid engraftment is important for platelet reconstitution after either myeloablative or busulfan-containing reduced intensity conditioning.
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