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Hematopoietic stem cell transplantation for infantile osteopetrosis.

Abstract：

:We report the international experience in outcomes after related and unrelated hematopoietic transplantation for infantile osteopetrosis in 193 patients. Thirty-four percent of transplants used grafts from HLA-matched siblings, 13% from HLA-mismatched relatives, 12% from HLA-matched, and 41% from HLA-mismatched unrelated donors. The median age at transplantation was 12 months. Busulfan and cyclophosphamide was the most common conditioning regimen. Long-term survival was higher after HLA-matched sibling compared to alternative donor transplantation. There were no differences in survival after HLA-mismatched related, HLA-matched unrelated, or mismatched unrelated donor transplantation. The 5- and 10-year probabilities of survival were 62% and 62% after HLA-matched sibling and 42% and 39% after alternative donor transplantation (P = .01 and P = .002, respectively). Graft failure was the most common cause of death, accounting for 50% of deaths after HLA-matched sibling and 43% of deaths after alternative donor transplantation. The day-28 incidence of neutrophil recovery was 66% after HLA-matched sibling and 61% after alternative donor transplantation (P = .49). The median age of surviving patients is 7 years. Of evaluable surviving patients, 70% are visually impaired; 10% have impaired hearing and gross motor delay. Nevertheless, 65% reported performance scores of 90 or 100, and in 17%, a score of 80 at last contact. Most survivors >5 years are attending mainstream or specialized schools. Rates of veno-occlusive disease and interstitial pneumonitis were high at 20%. Though allogeneic transplantation results in long-term survival with acceptable social function, strategies to lower graft failure and hepatic and pulmonary toxicity are urgently needed.

journal_name

Blood

journal_title

Blood

authors

Orchard PJ,Fasth AL,Le Rademacher J,He W,Boelens JJ,Horwitz EM,Al-Seraihy A,Ayas M,Bonfim CM,Boulad F,Lund T,Buchbinder DK,Kapoor N,O'Brien TA,Perez MA,Veys PA,Eapen M

doi

10.1182/blood-2015-01-625541

subject

Has Abstract

pub_date

2015-07-09 00:00:00

pages

270-6

issue

2

eissn

0006-4971

issn

1528-0020

pii

blood-2015-01-625541

journal_volume

126

pub_type

杂志文章

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