Cyclophosphamide-based in vivo T-Cell depletion for HLA-haploidentical transplantation in Fanconi anemia

Pediatric Hematology and Oncology

Volumn 29, Issue 6, 2012, Pages 568-578

  Thakar, M S ^a   Bonfim, C ^b   Sandmaier, B M ^c   O'Donnell, P ^c   Ribeiro, L ^b   Gooley, T ^c   Deeg, H J ^c   Flowers, M E ^c   Pasquini, R ^b   Storb, R ^c   Woolfrey, A E ^c   Kiem, H P ^c  

^a MEDICAL COLLEGE OF WISCONSIN   (United States)

^b FEDERAL UNIVERSITY OF PARANÁ   (Brazil)

^c Fred Hutchinson Cancer Research Center   (United States)

Author keywords

Aplastic anemia; Cyclophosphamide; Fanconi anemia; Haploidentical transplant

Indexed keywords

ALKALINE PHOSPHATASE; AMINOTRANSFERASE; BILIRUBIN; CD3 ANTIGEN; COTRIMOXAZOLE; CYCLOPHOSPHAMIDE; CYCLOSPORIN; FERRITIN; FLUDARABINE; GRANULOCYTE COLONY STIMULATING FACTOR; HLA A ANTIGEN; HLA B ANTIGEN; HLA C ANTIGEN; HLA DQB1 ANTIGEN; HLA DRB1 ANTIGEN; MESNA; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PREDNISONE; STEROID;

ABDOMINAL DISTENSION; ACINETOBACTER INFECTION; ACUTE GRAFT VERSUS HOST DISEASE; ADOLESCENT; ALKALINE PHOSPHATASE BLOOD LEVEL; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; AMINOTRANSFERASE BLOOD LEVEL; ANDROGEN THERAPY; ANOREXIA; APLASTIC ANEMIA; ARTICLE; AUTOPSY; BILIRUBIN BLOOD LEVEL; BONE MARROW DEPRESSION; BRACHYDACTYLY; CAFE AU LAIT SPOT; CASE REPORT; CAUSE OF DEATH; CHILD; CLINODACTYLY; COLONOSCOPY; COMPUTER ASSISTED TOMOGRAPHY; CYTOMEGALOVIRUS INFECTION; DIARRHEA; DISEASE SEVERITY; DONOR; DRUG DOSE REDUCTION; DRUG WITHDRAWAL; ENGRAFTMENT; ESOPHAGUS ATRESIA; FANCONI ANEMIA; FATIGUE; FEMALE; FERRITIN BLOOD LEVEL; FEVER; FOLLOW UP; FOOT INJURY; GRAFT REJECTION; HAPLOTYPE; HEMORRHAGIC CYSTITIS; HIRSUTISM; HLA SYSTEM; HORSESHOE KIDNEY; HUMAN; HYPERBILIRUBINEMIA; HYPERTELORISM; HYPERTRANSAMINASEMIA; IMMUNOSUPPRESSIVE TREATMENT; IN VIVO STUDY; IRON OVERLOAD; LUNG HEMORRHAGE; MORTALITY; MUCOSA INFLAMMATION; MULTIPLE ORGAN FAILURE; MULTIPLE PULMONARY NODULES; NAUSEA; NEUTROPENIA; NEUTROPHIL; PALLOR; PNEUMOCYSTIS PNEUMONIA; PRESCHOOL CHILD; QUALITY OF LIFE; RADIATION CHIMERA; RADIATION DOSE; RESPIRATORY FAILURE; RESPIRATORY TRACT INTUBATION; SCHOOL CHILD; SEPTIC SHOCK; SURVIVOR; T CELL DEPLETION; THORAX PAIN; THROMBOCYTE TRANSFUSION; THROMBOCYTOPENIA; THUMB MALFORMATION; TOXOPLASMOSIS; VIRUS REACTIVATION;

ADOLESCENT; ANTINEOPLASTIC AGENTS; CHILD; COMBINED MODALITY THERAPY; FANCONI ANEMIA; FEMALE; FOLLOW-UP STUDIES; GRAFT REJECTION; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HLA ANTIGENS; HUMANS; LYMPHOCYTE DEPLETION; T-LYMPHOCYTES; TRANSPLANTATION CHIMERA; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, HOMOLOGOUS; VIDARABINE;

EID: 84865314809     PISSN: 08880018     EISSN: 15210669     Source Type: Journal    
DOI: 10.3109/08880018.2012.708708     Document Type: Article

References (29)

1. Kutler DI, Singh B, Satagopan J, et al. A 20-year perspective on the international fanconi anemia registry (IFAR). Blood. 2003;101(4):1249-1256. (Pubitemid 36182492)
2. Wagner JE, Eapen M, MacMillan ML, et al. Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia. Blood. 2007;109(5):2256- 2262. (Pubitemid 46348231)
3. Thakar MS, Kurre P, Storb R, et al. Treatment of Fanconi anemia patients using fludarabine and lowdose TBI, followed by unrelated donorhematopoietic cell transplantation. Bone Marrow Transplant. 2011;46(4):539-544.
4. Chaudhury S, Auerbach AD, Kernan NA, et al. Fludarabine-based cytoreductive regimen and T-celldepleted grafts fromalternative donors for the treatment of high-risk patients with Fanconi anaemia (review). Br J Haematol. 2008;140(6):644-655. (Pubitemid 351300918)
5. Motwani J, Lawson SE, Darbyshire PJ. Successful HSCT using nonradiotherapy-based conditioning regimens and alternative donors in patients with Fanconi anaemia-experience in a single UK centre. Bone Marrow Transplant. 2005;36(5):405-410. (Pubitemid 43115027)
6. Rossi G, Giorgiani G, Comoli P, et al. Successful T-cell-depleted, related haploidentical peripheral blood stem cell transplantation in a patientwith Fanconi anaemia using a fludarabine-based preparative regimen without radiation. Bone Marrow Transplant. 2003;31(6):437-440. (Pubitemid 36511808)
7. O'Donnell PV, Luznik L, Jones RJ, et al. Nonmyeloablative bone marrow transplantation from partially HLA-mismatched related donors using posttransplantation cyclophosphamide. Biol Blood Marrow Transplant. 2002;8(7):377-386.
8. 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;14:641-650. (Pubitemid 351664058)
9. Luznik L, Bolanos-Meade J, Zahurak M, et al. High-dose cyclophosphamide as single-agent, shortcourse prophylaxis of graft-versus-host disease. Blood. 2010;115(16):3224-3230.
10. McSweeney PA, Niederwieser D, Shizuru JA, et al. Hematopoietic cell transplantation in older patients with hematologic malignancies: replacing high-dose cytotoxic therapy with graft-versustumor effects. Blood. 2001;97(11):3390-3400.
11. MarisMB, Niederwieser D, Sandmaier BM, et al. HLA-matched unrelated donor hematopoietic cell transplantation after nonmyeloablative conditioning for patients with hematologic malignancies. Blood. 2003;102(6):2021-2030. (Pubitemid 37122375)
12. Brodsky RA, Luznik L, Bolanos-Meade J, et al. Reduced intensity HLA-haploidentical BMT with post transplantation cyclophosphamide in nonmalignant hematologic diseases. Bone Marrow Transplant. 2008;42(8):523-527.
13. Bonfim CM, de Medeiros CR, Bitencourt MA, et al. HLA-matched related donor hematopoietic cell transplantation in43 patientswith fanconi anemia conditionedwith 60 mg/kg of cyclophosphamide. Biol Blood Marrow Transplant. 2007;13:1455-1460. (Pubitemid 350091381)
14. Zanis-Neto J, Flowers MED, Medeiros CR, et al. Low-dose cyclophosphamide conditioning for haematopoietic cell transplantation fromHLA-matched related donors in patientswith Fanconi anemia. Br J Haematol. 2005;130:99-106. (Pubitemid 40979140)
15. Glucksberg H, Storb R, Fefer A, et al. Clinical manifestations of graft-versus-host disease in human recipients ofmarrow from HL-A-matched sibling donors. Transplantation. 1974;18:295-304.
16. Sivgin S, Baldane S, Kaynar L, et al. Pretransplant serum ferritin levelmay be a predictivemarker for outcomes in patients having undergone allogeneic hematopoietic stem cell transplantation. Neoplasma. 2012;59(2):183-190.
17. Gluckman E, Auerbach AD, Horowitz MM, et al. Bone marrow transplantation for Fanconi anemia. Blood. 1995;86(7):2856-2862.
18. Guardiola P, Pasquini R, Dokal I, et al. Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European group for blood andmarrow transplantation. Blood. 2000;95(2):422-429. (Pubitemid 30041652)
19. MacMillan ML, Wagner JE. Haematopoeitic cell transplantation for Fanconi anaemia-when and how? (review). Br J Haematol. 2010;149(1):14-21.
20. Guardiola P, Socie G, Li X, et al. Acute graft-versus-host disease in patients with Fanconi anemia or acquired aplastic anemia undergoing bone marrow transplantation from HLA-identical sibling donors: risk factors and influence on outcome. Blood. 2004;103(1):73-77. (Pubitemid 38029920)
21. Masserot C, Peffault de Latour R, Rocha V, et al. Head and neck squamous cell carcinoma in 13 patients with Fanconi anemia after hematopoietic stem cell transplantation. Cancer. 2008;113(12):3315-3322.
22. Boulad F, Gillio A, Small TN, et al. Stem cell transplantation for the treatment of Fanconi anaemia using a fludarabine-based cytoreductive regimen and T-cell-depleted related HLA-mismatched peripheral blood stem cell grafts. Br J Haematol. 2000;111(4):1153-1157. (Pubitemid 32104932)
23. Dufort G, Pisano S, Incoronato A, et al. Feasibility and outcome of haploidentical SCT in pediatric high-risk hematologic malignancies and Fanconi anemia in Uruguay. Bone Marrow Transplant. 2012;47(5):663-668.
24. Locatelli F,ZeccaM,Pession A, et al.The outcome of childrenwith Fanconi anemia givenhematopoietic stem cell transplantation and the influence of fludarabine in the conditioning regimen: a report from the Italian pediatric group. Haematologica. 2007;92(10):1381-1388. (Pubitemid 350144156)
25. Luznik L, Jalla S, Engstrom LW, et al. Durable engraftment of major histocompatibility complexincompatible cells after nonmyeloablative conditioning with fludarabine, low-dose total body irradiation, and posttransplantation cyclophosphamide. Blood. 2001;98(12):3456-3464.
26. Santos GW, Tutschka PJ, Brookmeyer R, et al. Cyclosporine plus methylprednisolone versus cyclophosphamide plus methylprednisolone as prophylaxis for graft-versus-host disease: a randomized double-blind study in patients undergoing allogeneicmarrowtransplantation. Clin Transplant. 1987;1:21-28.
27. MacMillanML, Auerbach AD, Davies SM, et al. Haematopoietic cell transplantation in patients with Fanconi anaemia using alternate donors: results of a total body irradiation dose escalation trial. Br J Haematol. 2000;109(1):121-129. (Pubitemid 30340588)
28. Yabe H, Inoue H,Matsumoto M, et al. Allogeneic haematopoietic cell transplantation fromalternative donorswith a conditioning regimen of low-dose irradiation, fludarabine and cyclophosphamide in Fanconi anaemia. Br J Haematol. 2006;134(2):208-212. (Pubitemid 43898612)
29. MacMillanML, Auerbach AD,Wagner JE.Hematopoietic cell transplantation in Fanconi anemia patients with biallelic BRCA2mutations. Blood. 2004;104(11):776a, #2838 (abstract).