Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A Primary Immune Deficiency Treatment Consortium study.
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Grunebaum Eyal, Arnold Danielle E, Logan Brent, Parikh Suhag, Marsh Rebecca A, Griffith Linda M, Mallhi Kanwaldeep, Chellapandian Deepak, Lim Stephanie Si, Deal Christin L, Kapoor Neena, Murguía-Favela Luis, Falcone Emilia Liana, Prasad Vinod K, Touzot Fabien, Bleesing Jack J, Chandrakasan Shanmuganathan, Heimall Jennifer R, Bednarski Jeffrey J, Broglie Larisa A, Chong Hey Jin, Kapadia Malika, Prockop Susan, Dávila Saldaña Blachy J, Schaefer Edo, Bauchat Andrea L, Teira Pierre, Chandra Sharat, Parta Mark, Cowan Morton J, Dvorak Christopher C, Haddad Elie, Kohn Donald B, Notarangelo Luigi D, Pai Sung-Yun, Puck Jennifer M, Pulsipher Michael A, Torgerson Troy R, Malech Harry L, Kang Elizabeth M, Leiding Jennifer W
Аннотация
OBJECTIVES: We sought to study HCT for p47phox CGD in North America.
METHODS: Thirty patients with p47phox CGD who received allogeneic HCT at Primary Immune Deficiency Treatment Consortium centers since 1995 were included.
RESULTS: Residual oxidative activity was present in 66.7% of patients. In the year before HCT, there were 0.38 CGD-related infections per person-years. Inflammatory diseases, predominantly of the lungs and bowel, occurred in 36.7% of the patients. The median age at HCT was 9.1 years (range 1.5-23.6 years). Most HCTs (90%) were performed after using reduced intensity/toxicity conditioning. HCT sources were HLA-matched (40%) and -mismatched (10%) related donors or HLA-matched (36.7%) and -mismatched (13.3%) unrelated donors. CGD-related infections after HCT decreased significantly to 0.06 per person-years (P = .038). The frequency of inflammatory bowel disease and the use of steroids also decreased. The cumulative incidence of graft failure and second HCT was 17.9%. The 2-year overall and event-free survival were 92.3% and 82.1%, respectively, while at 5 years they were 85.7% and 77.0%, respectively. In the surviving patients evaluated, ≥95% donor myeloid chimerism at 1 and 2 years after HCT was 93.8% and 87.5%, respectively.
CONCLUSIONS: Patients with p47phox CGD suffer from a significant disease burden that can be effectively alleviated by HCT. Similar to other forms of CGD, HCT should be considered for patients with p47phox CGD.