Long-Term Follow-Up after Reduced-Intensity Conditioning and Stem Cell Transplantation for Childhood Nonmalignant Disorders

Biology of Blood and Marrow Transplantation

Volumn 22, Issue 8, 2016, Pages 1467-1472

  Madden, Lisa M ^a   Hayashi, Robert J ^a   Chan, Ka Wah ^b   Pulsipher, Michael A ^c   Douglas, Dorothea ^d   Hale, Gregory A ^e   Chaudhury, Sonali ^f   Haut, Paul ^g   Kasow, Kimberly A ^h   Gilman, Andrew L ⁱ   Murray, Lisa M ^a   Shenoy, Shalini ^a  

^a WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b Methodist Children's Hospital of South Texas   (United States)

^c UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^d PHOENIX CHILDREN'S HOSPITAL   (United States)

^e JOHNS HOPKINS ALL CHILDREN S HOSPITAL   (United States)

^f CHILDREN'S MEMORIAL HOSPITAL   (United States)

^g RILEY HOSPITAL FOR CHILDREN   (United States)

^h UNIVERSITY OF NORTH CAROLINA   (United States)

ⁱ Levine Children s Hospital   (United States)

Author keywords

Childhood nonmalignant disorders; Late complications; Reduced intensity conditioning; Stem cell transplantation

Indexed keywords

ALEMTUZUMAB; FLUDARABINE; MELPHALAN; MYELOABLATIVE AGENT; VIDARABINE;

ACADEMIC ACHIEVEMENT; ADOLESCENT; ADRENOLEUKODYSTROPHY; ADULT; APLASTIC ANEMIA; ARTICLE; AUTOIMMUNE HEMOLYTIC ANEMIA; AVASCULAR NECROSIS; BONE MARROW DEPRESSION; BRONCHIECTASIS; CD4 LYMPHOCYTE COUNT; CHILD; CHILDHOOD DISEASE; CHIMERA; CHRONIC GRAFT VERSUS HOST DISEASE; CORD BLOOD STEM CELL TRANSPLANTATION; CYTOMEGALOVIRUS; DELAYED PUBERTY; DONOR LYMPHOCYTE INFUSION; ENGRAFTMENT; ENTEROPATHY; FEMALE; FOLLOW UP; GLAUCOMA; GONAD FUNCTION; GRAFT SURVIVAL; HEART FUNCTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOGLOBINOPATHY; HUMAN; HURLER SYNDROME; HYPOTHYROIDISM; IMMUNE DEFICIENCY; IMMUNOSUPPRESSIVE TREATMENT; IPEX SYNDROME; KERATITIS; KIDNEY FUNCTION; LUNG FUNCTION; MAJOR CLINICAL STUDY; MALE; MELANOMA; MENORRHAGIA; METABOLIC DISORDER; MULTICENTER STUDY; MUSCULOSKELETAL FUNCTION; NEOPLASM; OVARY FUNCTION; OVARY INSUFFICIENCY; PERIPHERAL BLOOD STEM CELL TRANSPLANTATION; POLYENDOCRINOPATHY; REDUCED INTENSITY CONDITIONING; SCLERODERMA; SJOEGREN SYNDROME; THYROID FUNCTION; UVEITIS; VIRUS REACTIVATION; ANALOGS AND DERIVATIVES; BONE MARROW TRANSPLANTATION; CLINICAL TRIAL; MORTALITY; PRESCHOOL CHILD; PROCEDURES; STEM CELL TRANSPLANTATION; SURVIVAL ANALYSIS; TRANSPLANTATION CONDITIONING; YOUNG ADULT;

ADOLESCENT; ADULT; ALEMTUZUMAB; BONE MARROW TRANSPLANTATION; CHILD; CHILD, PRESCHOOL; FEMALE; FOLLOW-UP STUDIES; GRAFT SURVIVAL; HEMOGLOBINOPATHIES; HUMANS; MALE; MELPHALAN; MYELOABLATIVE AGONISTS; STEM CELL TRANSPLANTATION; SURVIVAL ANALYSIS; TRANSPLANTATION CONDITIONING; VIDARABINE; YOUNG ADULT;

EID: 84981313853     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2016.04.025     Document Type: Article

References (43)

1. 1 Shenoy, S., Grossman, W., DiPersio, J., et al. A novel reduced-intensity stem cell transplant regimen for non-malignant disorders. Bone Marrow Transplant 35 (2005), 345–352.
2. 2 Satwani, P., Cooper, N., Rao, K., et al. Reduced intensity conditioning and allogeneic stem cell transplantation in childhood malignant and nonmalignant diseases. Bone Marrow Transplantation 41 (2008), 173–182.
3. 3 Dvorak, C.C., Cowan, M., Hematopoietic stem cell transplantation for primary immunodeficiency disease. Bone Marrow Transplant 41 (2008), 119–126.
4. 4 Boelens, J., Prasad, V., Tolar, J., et al. Current international perspectives on hematopoietic stem cell transplantation for inherited metabolic disorders. Pediatr Clin North Am 57 (2010), 123–145.
5. 5 Locatelli, F., Kabbara, N., Ruggeri, A., et al. Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling. Blood 122 (2013), 1072–1078.
6. 6 Fagioli, F., Zecca, M., Lanino, E., et al. Haematopoietic stem cell transplantation for Diamond Blackfan anaemia: a report from the Italian Association of Paediatric Haematology and Oncology Registry. Br J Haematol 165 (2014), 673–681.
7. 7 Baker, K.S., Bresters, D., Sande, J.E., The burden of cure: long-term side effects following hematopoietic stem cell transplantation (HSCT) in children. Pediatr Clin North Am 57 (2010), 323–342.
8. 8 Bresters, D., van Gils, I.C.M., Kollen, W.J.W., et al. High burden of late effects after haematopoietic stem cell transplantation in childhood: a single-centre study. Bone Marrow Transplant 45 (2010), 79–85.
9. 9 Dahllof, G., Hingorani, S.R., Sanders, J.E., Late effects following hematopoietic cell transplantation for children. Biol Blood Marrow Transplant 14 (2008), 88–93.
10. 10 Hoffmeister, P.A., Madtes, D.K., Storer, B.E., Sanders, J.E., Pulmonary function in long-term survivors of pediatric hematopoietic cell transplantation. Pediatr Blood Cancer 47 (2006), 594–606.
11. 11 Majhail, N., Brazauskas, R., Rizzo, J., et al. Secondary solid cancers after allogeneic hematopoietic cell transplantation using busulfan-cyclophosphamide conditioning. Blood 117 (2011), 316–322.
12. 12 King, A., Kamani, N., Bunin, N., et al. Successful matched sibling donor marrow transplantation following reduced intensity conditioning in children with hemoglobinopathies. Am J Hematol 90 (2015), 1093–1098.
13. 13 Pai, S., Logan, B., Griffith, L., et al. Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med 371 (2014), 434–446.
14. 14 Parikh, S., Szabolcs, P., Reduced-intensity conditioning (RIC) in children with nonmalignant disorders (NMD) undergoing unrelated donor umbilical cord blood transplantation (UCBT). Biol Blood Marrow Transplant 18 (2012), S53–S55.
15. 15 Marsh, R., Kim, M., Liu, C., et al. An intermediate alemtuzumab schedule reduces the incidence of mixed chimerism following reduced-intensity conditioning hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis. Biol Blood Marrow Transplant 19 (2013), 1625–1631.
16. 16 Giralt, S., Bollen, K., Rizzo, D., et al. Reduced-intensity conditioning regimen workshop: defining the dose spectrum. Report of a workshop convened by the Center for International Blood and Marrow Transplant Research. Biol Blood Marrow Transplant 15 (2009), 367–369.
17. 17 National Cancer Institute Common Terminology Criteria for Adverse Events CTCAE 4.0. Available at: http://ctep.cancer.gov/protocolDevelopment/electronic_applications/ctc.htm. Last accessed January 2016.
18. 18 Wei, C., Crowne, E., Recent advances in the understanding and management of delayed puberty. Arch Dis Child 101 (2016), 481–488.
19. 19 Dirou, S., Malard, F., Chambellan, A., et al. Stable long-term pulmonary function after fludarabine, antithymocyte globulin and i.v. BU for reduced-intensity conditioning allogeneic SCT. Bone Marrow Transplant 49 (2014), 622–627.
20. 20 Armenian, S., Sun, C., Kawashima, T., et al. Long-term health-related outcomes in survivors of childhood cancer treated with HSCT versus conventional therapy: a report from the Bone Marrow Transplant Survivor Study (BMTSS) and Childhood Cancer Survivor Study (CCSS). Blood 118 (2011), 1413–1420.
21. 21 Faraci, M., Bekassy, A.N., DeFazio, V., et al. Non-endocrine late complications in children after allogeneic haematopoietic SCT. Bone Marrow Transplant 41 (2008), S49–S57.
22. 22 Uderzo, C., Pillon, M., Corti, P., et al. Impact of cumulative antracycline dose, preparative regimen and chronic graft-versus-host disease on pulmonary and cardiac function in children 5 years after allogeneic hematopoietic stem cell transplantation: a prospective evaluation on behalf of the EBMT Pediatric Diseases and Late Effects Working Parties. Bone Marrow Transplant 39 (2007), 667–675.
23. 23 Gower, W.A., Collaco, J.M., Mogayzel, P.J., Lung function and late pulmonary complications among survivors of hematopoietic stem cell transplantation during childhood. Pediatr Respir Rev 11 (2010), 115–122.
24. 24 Cohen, A., Bekassy, A., Gaiero, A., et al. Endocrinological late complications after hematopoietic SCT in children. Bone Marrow Transplant 41 (2008), S3–S8.
25. 25 Perkins, J.L., Kunin-Batson, A.S., Youngren, N.M., et al. Long-term follow-up of children who underwent hematopoeitic cell transplant (HCT) for AML or ALL at less than 3 years of age. Pediatr Blood Cancer 49 (2007), 958–963.
26. 26 Hows, J.M., Passweg, J.R., Tichelli, A., et al. Comparison of long-term outcomes after allogeneic hematopoietic stem cell transplantation from matched sibling and unrelated donors. Bone Marrow Transplant 38 (2006), 799–805.
27. 27 Kramer, J., Crittenden, M., DeSantes, K., Cowan, M., Cognitive and adaptive behavior 1 and 3 years following bone marrow transplantation. Bone Marrow Transplant 19 (1997), 607–613.
28. 28 Walters, M.C., Hardy, K., Edwards, S., et al. Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease. Biol Blood Marrow Transplant 16 (2010), 263–272.
29. 29 Eggleston, B., Patience, M., Edwards, S., et al. Effect of myeloablative bone marrow transplantation on growth in children with sickle cell anaemia: results of the multicenter study of haematopoietic cell transplantation for sickle cell anaemia. Br J Haematol 136 (2007), 673–676.
30. 30 Buchbinder, D., Nugent, D., Brazauskas, R., et al. Late effects in hematopoietic cell transplant recipients with acquired severe aplastic anemia: a report from the late effects working committee of the Center for International Blood and Marrow Transplant Research. Biol Blood Marrow Transplant 18 (2012), 1776–1784.
31. 31 Sanders, J., Woolfrey, A., Carpenter, P., et al. Late effects among pediatric patients followed for nearly 4 decades after transplantation for severe aplastic anemia. Blood 118 (2011), 1421–1428.
32. 32 Ringdén, O., Brazauskas, R., Wang, Z., et al. Second solid cancers after allogeneic hematopoietic cell transplantation using reduced-intensity conditioning. Biol Blood Marrow Transplant 20 (2014), 1777–1784.
33. 33 Mijovic, A., Abdallah, A., Pearce, L., et al. Effects on erythropoiesis of alemtuzumab-containing reduced intensity and standard conditioning regimens. Br J Haematol 142 (2008), 444–452.
34. 34 Daniels, G., Vladic, A., Brinar, V., et al. Alemtuzumab-related thyroid dysfunction in a phase 2 trial of patients with relapsing-remitting multiple sclerosis. J Clin Endocrinol Metab 99 (2014), 80–89.
35. 35 Afify, Z., Shaw, P.J., Clavano-Harding, A., Cowell, C.T., Growth and endocrine function in children with acute myeloid leukaemia after bone marrow transplantation using busulfan/cyclophosphamide. Bone Marrow Transplant 25 (2000), 1087–1092.
36. 36 Dvorak, C.C., Gracia, C.R., Sanders, J.E., et al. NCI, NHLBI/PBMTC first international conference on late effects after pediatric hematopoietic cell transplantation: endocrine challenges-thyroid dysfunction, growth impairment, bone health, & reproductive risks. Biol Blood Marrow Transplant 17 (2011), 1725–1738.
37. 37 King, A., Rodeghier, M., Panepinto, J., et al. Silent cerebral infarction, income, and grade retention among students with sickle cell anemia. Am J Hematol 89 (2014), E188–E192.
38. 38 Kupst, M., Penati, B., Debban, B., et al. Cognitive and psychosocial functioning of pediatric hematopoietic stem cell transplant patients: a prospective longitudinal study. Bone Marrow Transplant 30 (2002), 609–617.
39. 39 Phipps, S., Rai, S.N., Leung, W.-H., et al. Cognitive and academic consequences of stem-cell transplantation in children. J Clin Oncol 26 (2008), 2027–2033.
40. 40 Cool, V., Long-term neuropsychological risks in pediatric bone marrow transplant: what we do we know?. Bone Marrow Transplant 18 (1996), S45–S49.
41. 41 Parsons, S.K., Phipps, S., Sung, L., et al. NCI, NHLBI/PBMTC first international conference on late effects after pediatric hematopoietic cell transplantation: health-related quality of life, functional, and neurocognitive outcomes. Biol Blood Marrow Transplant 18 (2012), 162–171.
42. 42 Smedler, A., Winiarski, J., Neuropsychological outcome in very young hematopoietic SCT recipients in relation to pretransplant conditioning. Bone Marrow Transplant 42 (2008), 515–522.
43. 43 Kamani, N., Walters, M., Carter, S., et al. Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). Biol Blood Marrow Transplant 18 (2012), 1265–1272.