Bone marrow transplantation for thalassemia from alternative related donors: Improved outcomes with a new approach

Blood

Volumn 122, Issue 15, 2013, Pages 2751-2756

  Gaziev, Javid ^a   Marziali, Marco ^a   Isgrò, Antonella ^a   Sodani, Pietro ^a   Paciaroni, Katia ^a   Gallucci, Cristiano ^a   Andreani, Marco ^a   Testi, Manuela ^a   De Angelis, Gioia ^a   Alfieri, Cecilia ^a   Cardarelli, Luisa ^a   Ribersani, Michela ^a   Armiento, Daniele ^a   Lucarelli, Guido ^a  

^a TOR VERGATA UNIVERSITY HOSPITAL   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ACICLOVIR; ANTIFUNGAL AGENT; AZATHIOPRINE; BUSULFAN; COTRIMOXAZOLE; CYCLOPHOSPHAMIDE; CYCLOSPORIN; FLUDARABINE; HYDROXYUREA; METHOTREXATE; METHYLPREDNISOLONE; THIOTEPA; THYMOCYTE ANTIBODY;

ADOLESCENT; ADULT; ANTIBIOTIC PROPHYLAXIS; ANTIVIRAL THERAPY; ARTICLE; BONE MARROW TRANSPLANTATION; CAUSE OF DEATH; CHILD; CLINICAL ARTICLE; COMPARATIVE STUDY; CONTROLLED STUDY; CYTOMEGALOVIRUS INFECTION; DIARRHEA; DONOR; ENGRAFTMENT; EPSTEIN BARR VIRUS; FEMALE; GRAFT INFECTION; GRAFT REJECTION; GRAFT VERSUS HOST REACTION; GRAM NEGATIVE SEPSIS; HEART ARREST; HEMORRHAGIC CYSTITIS; HERPES VIRUS INFECTION; HLA MATCHING; HUMAN; HYPERTRANSAMINASEMIA; IMMUNOSUPPRESSIVE TREATMENT; INFANT; INFECTION PREVENTION; INTESTINAL BLEEDING; INTESTINE OBSTRUCTION; LIVER VEIN OBSTRUCTION; LOW DRUG DOSE; MALE; MATCHED SIBLING DONOR; MORBIDITY; NEUROTOXICITY; ORAL MUCOSITIS; OUTCOME ASSESSMENT; PATIENT SAFETY; PHENOTYPE; PNEUMOCYSTOSIS; PNEUMONIA; PRESCHOOL CHILD; PRIORITY JOURNAL; PROSPECTIVE STUDY; RELATED DONOR; SCHOOL CHILD; SEIZURE; STENOSIS; SURGICAL MORTALITY; SURVIVAL; SURVIVAL RATE; THALASSEMIA; TREATMENT DURATION; VIRUS REACTIVATION;

EID: 84888228928     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2013-07-513473     Document Type: Article

References (36)

1. Borgna-Pignatti C, Rugolotto S, De Stefano P, et al. Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine. Haematologica. 2004;89(10):1187-1193.
2. Piga A, Galanello R, Forni GL, et al. Randomized phase II trial of deferasirox (Exjade, ICL670), a once-daily, orally-administered iron chelator, in comparison to deferoxamine in thalassemia patients with transfusional iron overload. Haematologica. 2006;91(7):873-880.
3. Cunningham MJ, Macklin EA, Neufeld EJ, Cohen AR; Thalassemia Clinical Research Network. Complications of beta-thalassemia major in North America. Blood. 2004;104(1):34-39.
4. Lucarelli G, Gaziev J. Advances in the allogeneic transplantation for thalassemia. Blood Rev. 2008;22(2):53-63.
5. Lucarelli G, Isgro A, Sodani P, Gaziev J. Hematopoietic stem cell transplantation in thalassemia and sickle cell anemia. Cold Spring Harb Perspect Med. 2012;2(5):a011825.
6. Gaziev J, Lucarelli G. Allogeneic cellular gene therapy for hemoglobinopathies. Hematol Oncol Clin N Am. 2010;24(6):1145-1163.
7. Anasetti C, Etzioni R, Petersdorf EW, Martin PJ, Hansen JA. Marrow transplantation from unrelated volunteer donors. Annu Rev Med. 1995;46:169-179.
8. Jawdat DM, Al Saleh S, Sutton P, Al Anazi H, Shubaili A, Tamim H, Hajeer AH. Chances of finding an HLA-matched sibling: The Saudi experience. Biol Blood Marrow Transplant. 2009;15(10):1342-1344.
9. Shamsi T, Hashmi K, Adil S, et al. The stem cell transplant program in Pakistan-the first decade. Bone Marrow Transplant. 2008;42(Suppl 1):S114-S117.
10. Hamamy H. Consanguineous marriages: preconception consultation in primary health care settings. J Community Genet. 2012;3(3):185-192.
11. Elbjeirami WM, Abdel-Rahman F, Hussein AA. Probability of finding an HLA-matched donor in immediate and extended families: the Jordanian experience. Biol Blood Marrow Transplant. 2013;19(2):221-226.
12. Klein T, Yaniv I, Stein J, Narinsky R, Finkelstein Y, Garty BZ. Extended family studies for the identification of allogeneic stem cell transplant donors in Jewish and Arabic patients in Israel. Pediatr Transplant. 2005;9(1):52-55.
13. Ottinger H, Grosse-Wilde M, Schmitz A, Grosse-Wilde H. Immunogenetic marrow donor search for 1012 patients: a retrospective analysis of strategies, outcome and costs. Bone Marrow Transplant. 1994;14(suppl 4):S34-S38.
14. Ash RC, Horowitz MM, Gale RP, et al. Bone marrow transplantation from related donors other than HLA-identical siblings: effect of T cell depletion. Bone Marrow Transplant. 1991;7(6):443-452.
15. Szydlo R, Goldman JM, Klein JP, et al. Results of allogeneic bone marrow transplants for leukemia using donors other than HLA-identical siblings. J Clin Oncol. 1997;15(5):1767-1777.
16. Gaziev D, Galimberti M, Lucarelli G, et al. Bone marrow transplantation from alternative donors for thalassemia: HLA-phenotypically identical relative and HLA-nonidentical sibling or parent transplants. Bone Marrow Transplant. 2000;25(8):815-821.
17. Gaziev J, Sodani P, Lucarelli G, et al. Second hematopoietic SCT in patients with thalassemia recurrence following rejection of the first graft. Bone Marrow Transplant. 2008;42(6):397-404.
18. Gaziev J, Isgrò A, Marziali M, et al. Higher CD3 (+) and CD34 (+) cell doses in the graft increase the incidence of acute GVHD in children receiving BMT for thalassemia. Bone Marrow Transplant. 2012;47(1):107-114.
19. Glucksberg H, Storb R, Fefer A, et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors. Transplantation. 1974;18(4):295-304.
20. Filipovich AH, Weisdorf D, Pavletic S, et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant. 2005;11(12):945-956.
21. Kaplan EL, Meier P. Nonparametric estimation from incomplete observations. J Am Stat Assoc. 1958;53(282):457-481.
22. Gooley TA, Leisenring W, Crowley J, Storer BE. Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Stat Med. 1999;18(6):695-706.
23. Kanda Y. Investigation of the freely available easy-to-use software 'EZR' for medical statistics. Bone Marrow Transplant. 2013;48(3):452-458.
24. Ishak K, Baptista A, Bianchi L, et al. Histological grading and staging of chronic hepatitis. J Hepatol. 1995;22(6):696-699.
25. Anasetti C. Hematopoietic cell transplantation from HLA partially matched related donors. In: Thomas ED, Blume KG, Forman SJ, eds. Hematopoietic Cell Transplantation. Boston, MA: Blackwell Science; 1999:904-914.
26. Kanda Y, Chiba SH, Hirai H, et al. Allogeneic hematopoietic stem cell transplantation from family members other than HLA-identical siblings over the last decade (1991-2000). Blood. 2003;102(4):1541-1547.
27. Shaw PJ, Kan F, Woo Ahn K, et al. Outcomes of pediatric bone marrow transplantation for leukemia and myelodysplasia using matched sibling, mismatched related, or matched unrelated donors. Blood. 2010;116(19):4007-4015.
28. Ottinger HD, Ferencik S, Beelen DW, et al. Hematopoietic stem cell transplantation: contrasting the outcome of transplantations from HLA-identical siblings, partially HLA-mismatched related donors, and HLA-matched unrelated donors. Blood. 2003;102(3):1131-1137.
29. Valcárcel D, Sierra J, Wang T, et al. One-antigen mismatched related versus HLA-matched unrelated donor hematopoietic stem cell transplantation in adults with acute leukemia: Center for International Blood and Marrow Transplant Research results in the era of molecular HLA typing. Biol Blood Marrow Transplant. 2011;17(5):640-648.
30. Sullivan KM, Anasetti C, Horowitz M, et al. Unrelated and HLA-nonidentical related donor marrow transplantation for thalassemia and leukaemia. Ann N Y Acad Sci. 1998;850:312-324.
31. Galambrun C, Pondarré C, Bertrand Y, et al; French Rare Disease Center for Thalassemia; French Society of Bone Marrow Transplantation. French multicenter 22-year experience in stem cell transplantation for beta-thalassemia major: lessons and future directions. Biol Blood Marrow Transplant. 2013;19(1):62-68.
32. Bacigalupo A. Matched and mismatched unrelated donor transplantation: is the outcome the same as for matched sibling donor transplantation? Hematology Am Soc Hematol Educ Program. 2012, 2012:223-229.
33. Petersdorf EW, Gooley T, Malkki M, et al. The biological significance of HLA-DP gene variation in haematopoietic cell transplantation. Br J Haematol. 2001;112(4):988-994.
34. Fleischhauer K, Locatelli F, Zecca M, et al. Graft rejection after unrelated donor hematopoietic stem cell transplantation for thalassemia is associated with nonpermissive HLA-DPB1 disparity in host-versus-graft direction. Blood. 2006;107(7):2984-2992.
35. Gallardo D, Brunet S, Torres A, et al. Hla-DPB1 mismatch in HLA-A-B-DRB1 identical sibling donor stem cell transplantation and acute graftversus-host disease. Transplantation. 2004;77(7):1107-1110.
36. Gaziev J, Nguyen L, Puozzo C, et al. Novel pharmacokinetic behavior of intravenous busulfan in children with thalassemia undergoing hematopoietic stem cell transplantation: a prospective evaluation of pharmacokinetic and pharmacodynamic profile with therapeutic drug monitoring. Blood. 2010;115(22):4597-4604.