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Titolo:
OUTCOME OF UNRELATED DONOR BONE-MARROW TRANSPLANTATION IN 40 CHILDRENWITH HURLER-SYNDROME
Autore:
PETERS C; BALTHAZOR M; SHAPIRO EG; KING RJ; KOLLMAN C; HEGLAND JD; HENSLEEDOWNEY J; TRIGG ME; COWAN MJ; SANDERS J; BUNIN N; WEINSTEIN H; LENARSKY C; FALK P; HARRIS R; BOWEN T; WILLIAMS TE; GRAYSON GH; WARKENTIN P; SENDER L; COOL VA; CRITTENDEN M; PACKMAN S; KAPLAN P; LOCKMAN LA; ANDERSON J; KRIVIT W; DUSENBERY K; WAGNER J;
Indirizzi:
UNIV IOWA,DEPT PEDIAT IOWA CITY IA 52242
Titolo Testata:
Blood
fascicolo: 11, volume: 87, anno: 1996,
pagine: 4894 - 4902
SICI:
0006-4971(1996)87:11<4894:OOUDBT>2.0.ZU;2-5
Fonte:
ISI
Lingua:
ENG
Soggetto:
VERSUS-HOST DISEASE; LYMPHOCYTES-T; SELECTIVE DEPLETION; CLINICAL-TRIAL; CHRONIC GRAFT; RESOLUTION; RECIPIENTS; LEUKEMIA; BUSULFAN; PROGRAM;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Science Citation Index Expanded
Citazioni:
57
Recensione:
Indirizzi per estratti:
Citazione:
C. Peters et al., "OUTCOME OF UNRELATED DONOR BONE-MARROW TRANSPLANTATION IN 40 CHILDRENWITH HURLER-SYNDROME", Blood, 87(11), 1996, pp. 4894-4902

Abstract

Long-term survival and improved neuropsychological function have occurred in selected children with Hurler syndrome (MPS I H) after successful engraftment with genotypically matched sibling bone marrow transplantation (BMT). However, because few children have HLA-identical siblings, the feasibility of unrelated donor (URD) BMT as a vehicle for adoptive enzyme therapy was evaluated in this retrospective study. Forty consecutive children (median, 1.7 years; range, 0.9 to 3.2 years) withMPS I H received high-dose chemotherapy with or without radiation followed by BMT between January 27, 1989 and May 13, 1994. Twenty-five ofthe 40 patients initially engrafted. An estimated 49% of patients arealive at 2 years, 63% alloengrafted and 37% autoengrafted. The probability of grade II to IV acute graft-versus-host disease (GVHD) was 30%, and the probability of extensive chronic GVHD was 18%. Eleven patients received a second URD BMT because of graft rejection or failure. Ofthe 20 survivors, 13 children have complete donor engraftment, two children have mixed chimeric grafts, and five children have autologous marrow recovery. The BM cell dose was correlated with both donor engraftment and survival. Thirteen of 27 evaluable patients were engrafted at 1 year following URD BMT. Neither T-lymphocyte depletion (TLD) of the bone marrow nor irradiation appeared to influence the likelihood of engraftment. Ten of 16 patients alive at 1 year who received a BM celldose greater than or equal to 3.5 x 10(8) cells/kg engrafted, and 62%are estimated to be alive at 3 years. In contrast, only 3 of 11 patients receiving less than 3.5 x 10(8) cells/kg engrafted, and 24% are estimated to be alive at 3 years (P = .05). The mental developmental index (MDI) was assessed before BMT. Both baseline and post-BMT neuropsychological data were available for 11 engrafted survivors. Eight children with a baseline MDI greater than 70 have undergone URD BMT (median age, 1.5 years; range, 1.0 to 2.4 years). Of these, two children have had BMT too recently for developmental follow-up. Of the remaining six, none has shown any decline in age equivalent scores. Four children are acquiring skills at a pace equal to or slightly below their same age peers; two children have shown a plateau in learning or extreme slowing in their learning process. For children with a baseline MDI less than 70 (median age, 2.5 years; range, 0.9 to 2.9 years), post-BMT follow-up indicated that two children have shown deterioration in their developmental skills. the remaining three children are maintaining theirskills and are adding to them at a highly variable rate. We conclude that MPS I H patients with a baseline MDI greater than 70 who are engrafted survivors following URD BMT can achieve a favorable long-term outcome and improved cognitive function. Future protocols must address the high risk of graft rejection or failure and the impact of GVHD in this patient population. (C) 1996 by The American Society of Hematology.

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Documento generato il 07/07/20 alle ore 22:37:27