Reduced Intensity Transplantation for Primary Immunodeficiency Disorders

Immunology and Allergy Clinics of North America

Volumn 30, Issue 1, 2010, Pages 103-124

  Veys, Paul ^a,b  

^a Department of BMT ^*  (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

Hematopoietic cell transplantation; Primary immunodeficiency; Reduced intensity

Indexed keywords

ALEMTUZUMAB; BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; ETOPOSIDE; FLUDARABINE; IMMUNOGLOBULIN; MELPHALAN; MONOCLONAL ANTIBODY; MONOCLONAL ANTIBODY CD45; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; OKT 3; RITUXIMAB; THIOTEPA; THYMOCYTE ANTIBODY; TREOSULFAN; UNCLASSIFIED DRUG;

AREA UNDER THE CURVE; CAPILLARY LEAK SYNDROME; CHIMERA; CHRONIC GRANULOMATOUS DISEASE; CLINICAL TRIAL; COMORBIDITY; CYTOMEGALOVIRUS INFECTION; DISEASE SEVERITY; DONOR LYMPHOCYTE INFUSION; DRUG BLOOD LEVEL; DRUG DOSE REDUCTION; DRUG MECHANISM; DRUG WITHDRAWAL; ENGRAFTMENT; GASTROINTESTINAL TOXICITY; GRAFT REJECTION; GRAFT VERSUS HOST REACTION; GROWTH RETARDATION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS; HUMAN; IMMUNE DEFICIENCY; INFERTILITY; LEUKOCYTE ADHESION DEFICIENCY; LIVER DISEASE; LOW DRUG DOSE; LUNG DISEASE; MINIMUM INHIBITORY CONCENTRATION; MYELOABLATIVE CONDITIONING; NONHUMAN; PRIORITY JOURNAL; PULMONARY HYPERTENSION; REDUCED INTENSITY CONDITIONING; REVIEW; RISK REDUCTION; SEVERE COMBINED IMMUNODEFICIENCY; SHOCK; SIDE EFFECT; SKIN TOXICITY; STEADY STATE; THROMBOSIS; VIRUS REACTIVATION; WHOLE BODY RADIATION; WISKOTT ALDRICH SYNDROME;

HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; IMMUNOLOGIC DEFICIENCY SYNDROMES; TRANSPLANTATION CONDITIONING; TREATMENT OUTCOME;

EID: 74849103303     PISSN: 08898561     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.iac.2009.11.003     Document Type: Review

References (56)

1. Gatti R.A., Meuwissen H.J., Allen H.D., et al. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet 2 7583 (1968) 1366-1369
2. Bach F.H., Albertini R.J., Joo P., et al. Bone-marrow transplantation in a patient with the Wiskott-Aldrich syndrome. Lancet 2 7583 (1968) 1364-1366
3. Filipovich A. Hematopoietic cell transplantation for correction of primary immunodeficiencies [review]. Bone Marrow Transplant 42 Suppl 1 (2008) S49-S52
4. Alousi A., and de Lima M. Reduced-intensity conditioning allogeneic hematopoietic stem cell transplantation. Clin Adv Hematol Oncol 5 (2007) 560-570
5. Barrett A.J., and Savani B.N. Stem cell transplantation with reduced-intensity conditioning regimens: a review of ten years experience with new transplant concepts and new therapeutic agents. Leukemia 20 (2006) 1661-1672
6. Rezvani A.R., and Storb R. Using allogeneic stem cell/T-cell grafts to cure hematologic malignancies. Expert Opin Biol Ther 8 (2008) 161-179
7. Eapen M., Horowitz M.M., Klein J.P., et al. Higher mortality after allogeneic peripheral-blood transplantation compared with bone marrow in children and adolescents: the Histocompatibility and Alternate Stem Cell Source Working Committee of the International Bone Marrow Transplant Registry. J Clin Oncol 22 (2004) 4872-4880
8. Pulsipher M.A., Nagler A., Iannone R., et al. Weighing the risks of G-CSF administration, leukopheresis, and standard marrow harvest: ethical and safety considerations for normal pediatric hematopoietic cell donors. Pediatr Blood Cancer 46 (2006) 422-433
9. Pulsipher M.A., Boucher K.M., Wall D., et al. Reduced-intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: results of the Pediatric Blood and Marrow Transplant Consortium Study ONC0313. Blood 114 7 (2009) 1429-1436
10. Iannone R., Casella J.F., Fuchs E.J., et al. Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant 9 (2003) 519-528
11. Woolfrey A., Pulsipher M.A., and Storb R. Nonmyeloablative hematopoietic cell transplant for treatment of immune deficiency [review]. Curr Opin Pediatr 13 6 (2001) 539-545
12. Satwani P., Cooper N., Rao K., et al. Reduced intensity conditioning and allogeneic stem cell transplantation in childhood malignant and nonmalignant diseases [review]. Bone Marrow Transplant 41 2 (2008) 173-182
13. Slavin S., Nagler A., Naparstek E., et al. Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases. Blood 91 3 (1998) 756-763
14. Champlin R., Khouri I., Shimoni A., et al. Harnessing graft-versus-malignancy: non-myeloablative preparative regimens for allogeneic haematopoietic transplantation, an evolving strategy for adoptive immunotherapy. Br J Haematol 111 (2000) 18-29
15. Storb R., Yu C., Wagner J.L., et al. Stable mixed hematopoietic chimerism in DLA-identical littermate dogs given sublethal total body irradiation before and pharmacological immunosuppression after marrow transplantation. Blood 89 8 (1997) 3048-3054
16. Giralt S., Estey E., Albitar M., et al. Engraftment of allogeneic hematopoietic progenitor cells with purine analog-containing chemotherapy: harnessing graft-versus-leukemia without myeloablative therapy. Blood 89 (1997) 4531-4536
17. Amrolia P., Gaspar B., Hassan A., et al. Non-myeloablative stem cell transplantation for congenital immunodeficiencies. Blood 96 (2000) 1239-1246
18. Rao K., Amrolia P.J., Jones A., et al. Improved survival after unrelated donor bone marrow transplant in children with primary immunodeficiency using a reduced intensity conditioning regimen. Blood 105 (2005) 879-885
19. Burroughs L.M., Storb R., Leisenring W.M., et al. Intensive post-grafting immune suppression combined with nonmyeloablative conditioning for transplantation of HLA-identical hematopoietic cell grafts: results of a pilot study for treatment of primary immunodeficiency disorders. Bone Marrow Transplant 40 (2007) 633-642
20. Qasim W., Cavazzana-Calvo M., Davies E.G., et al. Allogeneic hematopoietic stem-cell transplantation for leukocyte adhesion deficiency. Pediatrics 123 3 (2009) 836-840
21. Bradley M.B., Satwani P., Baldinger L., et al. Reduced intensity allogeneic umbilical cord blood transplantation in children and adolescent recipients with malignant and non-malignant diseases. Bone Marrow Transplant 40 7 (2007) 621-631
22. Barker J.N., Weisdorf D.J., DeFor T.E., et al. Rapid and complete donor chimerism in adult recipients of unrelated donor umbilical cord blood transplantation after reduced-intensity conditioning. Blood 102 5 (2003) 1915-1919
23. Jacobsohn D.A., Duerst R., Tse W., et al. Reduced intensity haemopoietic stem-cell transplantation for treatment of non-malignant diseases in children. Lancet 364 (2004) 156-162
24. Cooper N., Rao K., Goulden N., et al. The use of reduced-intensity stem cell transplantation in haemophagocytic lymphohistiocytosis and Langerhans cell histiocytosis. Bone Marrow Transplant 42 Suppl 2 (2008) S47-S50
25. Cooper N., Rao K., Gilmour K., et al. Stem cell transplantation with reduced intensity conditioning for haemophagocytic lymphohistiocytosis. Blood 107 3 (2006) 1233-1236
26. Ochs H.D., Filipovich A.H., Veys P., et al. Wiskott-Aldrich syndrome: diagnosis, clinical and laboratory manifestations, and treatment. Biol Blood Marrow Transplant 15 Suppl 1 (2008) 84-90
27. Horn B., Baxter-Lowe L.A., Englert L., et al. Reduced intensity conditioning using intravenous busulfan, fludarabine and rabbit ATG for children with nonmalignant disorders and CML. Bone Marrow Transplant 37 3 (2006) 263-269
28. Straathof K.C., Rao K., Eyrich M., et al. Haemopoietic stem cell transplantation with antibody-based minimal intensity conditioning regimen for children with severe organ toxicity or DNA repair disorders. Lancet 374 (2009) 912-920
29. Greystoke B., Bonanomi S., Carr T.F., et al. Treosulfan-containing regimens achieve high rates of engraftment associated with low transplant morbidity and mortality in children with non-malignant disease and significant co-morbidities. Br J Haematol 142 (2008) 257-262
30. Seger R.A. Modern management of chronic granulomatous disease. Br J Haematol 140 3 (2008) 255-266
31. Ozyurek E., Cowan M.J., Koerper M.A., et al. Increasing mixed chimerism and the risk of graft loss in children undergoing allogeneic hematopoietic stem cell transplantation for non-malignant disorders. Bone Marrow Transplant 42 2 (2008) 83-91
32. Veys P., Rao K., and Amrolia P. Stem cell transplantation for congenital immunodeficiencies using reduced-intensity conditioning. Bone Marrow Transplant 35 Suppl 1 (2005) S45-S47
33. Cohen J., Gandi M., Naik P., et al. Increased incidence of EBV-related disease following paediatric stem cell transplantation with reduced-intensity conditioning. Br J Haematol 129 2 (2005) 229-239
34. Cohen J.M., Sebire N.J., Harvey J., et al. Successful treatment of lymphoproliferative disease complicating primary immunodeficiency/immunodysregulatory disorders with reduced-intensity allogeneic stem cell transplantation. Blood 110 6 (2007) 2209-2214
35. Chakrabarti S., Mackinnon S., Chopra R., et al. High incidence of cytomegalovirus infection after nonmyeloablative stem cell transplantation: potential role of Campath-1H in delaying immune reconstitution. Blood 99 12 (2002) 4357-4363
36. Shenoy S., Grossman W.J., DiPersio J., et al. A novel reduced-intensity stem cell transplant regimen for nonmalignant disorders. Bone Marrow Transplant 35 4 (2005) 345-352
37. Sobecks R.M., Ball E.J., Askar M., et al. Influence of killer immunoglobulin-like receptor/HLA ligand matching on achievement of T-cell complete donor chimerism in related donor nonmyeloablative allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplant 41 8 (2008) 709-714
38. Casper J., Knauf W., Blau I., et al. Treosulfan/fludarabine: a new conditioning regimen in allogeneic transplantation. Ann Hematol 83 Suppl 1 (2004) S70-S71
39. Casper J., Knauf W., Kiefer T., et al. Treosulfan and fludarabine: a new toxicity-reduced conditioning regimen for allogeneic hematopoietic stem cell transplantation. Blood 103 (2004) 725-731
40. Beelen D.W., Trenschel R., Casper J., et al. Dose-escalated treosulphan in combination with cyclophosphamide as a new preparative regimen for allogeneic haematopoietic stem cell transplantation in patients with an increased risk for regimen-related complications. Bone Marrow Transplant 35 (2005) 233-241
41. Matthes-Martin S., Lion T., Hass O.A., et al. Lineage-specific chimaerism after stem cell transplantation in children following reduced intensity conditioning: potential predictive value of NK cell chimaerism for late graft rejection. Leukemia 17 (2003) 1934-1942
42. Gennery A.R., and Cant A.J. Cord blood stem cell transplantation in primary immune deficiencies [review]. Curr Opin Allergy Clin Immunol 7 6 (2007) 528-534
43. Chen Y.B., and Spitzer T.R. Current status of reduced-intensity allogeneic stem cell transplantation using alternative donors. Leukemia 22 1 (2008) 31-41
44. Antoine C., Muller S., Cant A., et al. Long term survival and haematopoietic stem cell transplantation for immunodeficiencies: a survey of the European experience 1968-1999. Lancet 361 (2003) 553-560
45. Horne A., Janka G., Maarten Egeler R., et al. Haematopoietic stem cell transplantation in haemophagocytic lymphohistiocytosis. Br J Haematol 129 (2005) 622-630
46. Vaughn G, Bleesing J, Jordan M, et al. Hematopoietic cell transplantation with reduced intensity conditioning (RIC HCT) for hemophagocytic lymphohistiocytosis (HLH) and X-linked lymphoproliferative syndrome [abstract]. Proceedings of the Histiocyte Society Meeting, Cambridge, September 2007. p. 20.
47. Ozsahin H., Cavazzana-Calvo M., Notarangelo L.D., et al. Long-term outcome following hematopoietic stem cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and the European Group for Blood and Marrow Transplantation. Blood 111 1 (2008) 439-445
48. Filipovich A.H., Stone J.V., Tomany S.C., et al. Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program. Blood 97 6 (2001) 1598-1603
49. Pai S.Y., DeMartiis D., Forino C., et al. Stem cell transplantation for the Wiskott-Aldrich syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation. Bone Marrow Transplant 38 (2006) 671-679
50. Horwitz M.E., Barrett A.J., Brown M.R., et al. Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft. N Engl J Med 344 12 (2001) 881-888
51. Güngör T., Halter J., Klink A., et al. Successful low toxicity hematopoietic stem cell transplantation for high-risk adult chronic granulomatous disease patients. Transplantation 79 11 (2005) 1596-1606
52. Gungor T., Halter J., Stussi G., et al. Successful busulphan-based reduced intensity conditioning in high-risk paediatric and adult chronic granulomatous disease - The Swiss experience [abstract]. Bone Marrow Transplant 43 Suppl 1 (2009) S75
53. Suzuki N., Hatakeyama N., Yamamoto M., et al. Treatment of McLeod phenotype chronic granulomatous disease with reduced-intensity conditioning and unrelated-donor umbilical cord blood transplantation. Int J Hematol 85 1 (2007) 70-72
54. Satwani P., Morris E., Bradley M.B., et al. Reduced intensity and non-myeloablative allogeneic stem cell transplantation in children and adolescents with malignant and non-malignant diseases [review]. Pediatr Blood Cancer 50 1 (2008) 1-8
55. Burkholder T.H., Colenda L., Tuschong L.M., et al. Reproductive capability in dogs with canine leukocyte adhesion deficiency treated with nonmyeloablative conditioning prior to allogeneic hematopoietic stem cell transplantation. Blood 108 5 (2006) 1767-1769
56. Titman P., Pink E., Skucek E., et al. Cognitive and behavioural abnormalities in children following haematopoietic stem cell transplantation for severe congenital immunodeficiencies. Blood 112 9 (2008) 3907-3913