HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease

Blood

Volumn 120, Issue 22, 2012, Pages 4285-4291

  Bolaños Meade, Javier ^a   Fuchs, Ephraim J ^a   Luznik, Leo ^a   Lanzkron, Sophie M ^b   Gamper, Christopher J ^a   Jones, Richard J ^a   Brodsky, Robert A ^a,b  

^a JOHNS HOPKINS UNIVERSITY   (United States)

^b JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CYCLOPHOSPHAMIDE; ETIRACETAM; FLUDARABINE; GRANULOCYTE COLONY STIMULATING FACTOR; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PENICILLIN K; PENICILLIN V; QUINOLONE; RAPAMYCIN; RECOMBINANT GRANULOCYTE COLONY STIMULATING FACTOR; RITUXIMAB; TACROLIMUS; THYMOCYTE ANTIBODY;

ACUTE GRAFT VERSUS HOST DISEASE; ADOLESCENT; ADULT; AGED; ALLOGENIC BONE MARROW TRANSPLANTATION; ARTICLE; CHILD; CLINICAL ARTICLE; DRUG MEGADOSE; EPSTEIN BARR VIRUS INFECTION; FEMALE; FOLLOW UP; GRAFT VERSUS HOST REACTION; HAPLOTYPE; HLA MATCHING; HUMAN; MALE; NONMYELOABLATIVE CONDITIONING; ORGAN DONOR; POSTERIOR REVERSIBLE ENCEPHALOPATHY SYNDROME; PRESCHOOL CHILD; PRIORITY JOURNAL; SCHOOL CHILD; SICKLE CELL ANEMIA; WHOLE BODY RADIATION;

EID: 84869813639     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2012-07-438408     Document Type: Article

References (32)

1. Lorey FW, Arnopp J, Cunningham GC. Distribution of hemoglobinopathy variants by ethnicity in a multiethnic state. Genet Epidemiol. 1996;13(5):501-512.
2. Powars DR, Chan LS, Hiti A, Ramicone E, Johnson C. Outcome of sickle cell anemia: a 4-decade observational study of 1056 patients. Medicine. 2005;84(6):363-376.
3. Hulbert ML, McKinstry RC, Lacey JL, et al. Silent cerebral infarcts occur despite regular blood transfusion therapy after first strokes in children with sickle cell disease. Blood. 2011;117(3):772-779.
4. Bernaudin F, Socié G, Kuentz M, et al. Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood. 2007;110(7):2749-2756.
5. Walters MC, Patience M, Leisenring W, et al. Bone marrow transplantation for sickle cell disease. N Engl J Med. 1996;335(6):369-376.
6. Jacobsohn DA, Duerst R, Tse W, Kletzel M. Reduced intensity haemopoietic stem-cell transplantation for treatment of non-malignant diseases in children. Lancet. 2004;364(9429):156-162.
7. Iannone R, Casella JF, Fuchs EJ, et al. Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant. 2003; 9(8):519-528.
8. Hsieh MM, Kang EM, Fitzhugh CD, et al. Allogeneic hematopoietic stem-cell transplantation for sickle cell disease. N Engl J Med. 2009;361(24):2309-2317.
9. Dew A, Collins D, Artz A, et al. Paucity of HLAidentical unrelated donors for African-Americans with hematologic malignancies: the need for new donor options. Biol Blood Marrow Transplant. 2008;14(8):938-941.
10. Walters MC, Sullivan KM. Stem-cell transplantation for sickle cell disease. N Engl J Med. 2010; 362(10):955-956.
11. Hsieh MM, Fitzhugh CD, Tisdale JF. Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now. Blood. 2011;118(5):1197-1207.
12. Bolaños-Meade J, Brodsky RA. Blood and marrow transplantation for sickle cell disease: overcoming barriers to success. Curr Opin Oncol. 2009;21(2):158-161.
13. Brodsky RA, Luznik L, Bolaños-Meade J, Leffell MS, Jones RJ, Fuchs EJ. Reduced intensity HLA-haploidentical BMT with post transplantation cyclophosphamide in nonmalignant hematologic diseases. Bone Marrow Transplant. 2008;42(8):523-527.
14. Luznik L, O'Donnell PV, Symons HJ, et al. HLA-haploidentical bone marrow transplantation for hematologic malignancies using nonmyeloablative conditioning and high-dose, posttransplantation cyclophosphamide. Biol Blood Marrow Transplant. 2008;14(6):641-650.
15. Luznik L, Jalla S, Engstrom LW, Iannone R, Fuchs EJ. Durable engraftment of major histocompatibility complex-incompatible cells after nonmyeloablative conditioning with fludarabine, low-dose total body irradiation, and posttransplantation cyclophosphamide. Blood. 2001; 98(12):3456-3464.
16. Emadi A, Jones RJ, Brodsky RA. Cyclophosphamide and cancer: golden anniversary. Nat Rev Clin Oncol. 2009;6(11):638-647.
17. Luznik L, Jones RJ, Fuchs EJ. High-dose cyclophosphamide for graft-versus-host disease prevention. Curr Opin Hematol. 2010;17(6):493-499.
18. Brunstein CG, Fuchs EJ, Carter SL, et al. Alternative donor transplantation after reduced intensity conditioning: results of parallel phase 2 trials using partially HLA-mismatched related bone marrow or unrelated double umbilical cord blood grafts. Blood. 2011;118(2):282-288.
19. Vermylen C. Hematopoietic stem cell transplantation in sickle cell disease. Blood Rev. 2003;17(3):163-166.
20. Powars D, Weidman JA, Odom-Maryon T, Niland JC, Johnson C. Sickle cell chronic lung disease: prior morbidity and the risk of pulmonary failure. Medicine. 1988;67(1):66-76.
21. 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.
22. Shaw PJ, Kan F, Woo AK, 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.
23. Brachet C, Heinrichs C, Tenoutasse S, Devalck C, Azzi N, Ferster A. Children with sickle cell disease: growth and gonadal function after hematopoietic stem cell transplantation. J Pediatr Hematol Oncol. 2007;29(7):445-450.
24. Walters MC, Sullivan KM, Bernaudin F, et al. Neurologic complications after allogeneic marrow transplantation for sickle cell anemia. Blood. 1995;85(4):879-884.
25. Baron F, Baker JE, Storb R, et al. Kinetics of engraftment in patients with hematologic malignancies given allogeneic hematopoietic cell transplantation after nonmyeloablative conditioning. Blood. 2004;104(8):2254- 2262.
26. McSweeney PA, Storb R. Mixed chimerism: preclinical studies and clinical applications. Biol Blood Marrow Transplant. 1999;5(4):192-203.
27. Carvallo C, Geller N, Kurlander R, et al. Prior chemotherapy and allograft CD34+ dose impact donor engraftment following nonmyeloablative allogeneic stem cell transplantation in patients with solid tumors. Blood. 2004;103(4):1560-1563.
28. Raj K, Streetly M, Kazmi M, et al. Peripheral blood progenitor cell (Pbpc) transplantation from haploidentical donors following reduced intensity conditioning (Ric) for high-risk hematologic malignancies. Biol Blood Marrow Transplant. 2012; 18(2):S331.
29. Chang YJ, Huang XJ. Use of G-CSF-stimulated marrow in allogeneic hematopoietic stem cell transplantation settings: a comprehensive review. Clin Transplant. 2011;25(1):13-23.
30. Montgomery RA, Lonze BE, King KE, et al. Desensitization in HLA-incompatible kidney recipients and survival. N Engl J Med. 2011;365(4):318-326.
31. Gladstone DE, Zachary A, Fuchs EJ, et al. Desensitization for mismatched hematopoietic stem cell transplantation (HSCT) [abstract]. Blood (ASH Annual Meeting Abstracts). 2011;118(21):1955.
32. Shenoy S. Hematopoietic stem cell transplantation for sickle cell disease: current practice and emerging trends. Hematology Am Soc Hematol Educ Program. 2011;2011:273-279.