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Pretransplantation: Indications and Timing

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Hematopoietic Stem Cell Transplantation for the Pediatric Hematologist/Oncologist

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Abstract

Hematopoietic stem cell transplantation (HSCT) is now indicated in over 100 disease types and subtypes. While autologous stem cell transplantation is mainly reserved for high-risk malignancy, allogeneic stem cell transplantation can be used in both malignant and nonmalignant disorders. The most common types of malignant disorders that undergo treatment with transplantation include leukemia, lymphoma, brain tumors, and neuroblastoma. The most common types of nonmalignant diseases that are treated with transplantation include severe aplastic anemia, inherited bone marrow failure syndromes, immunodeficiencies, hemoglobinopathies, and inherited metabolic disorders. For nearly all indications, chemotherapy is given prior to the transplant either as primary treatment for the disease and/or to allow space for the new stem cells through myeloablation. Toxicity from both the chemotherapy as well as infectious risks should be balanced with the existing clinical status of the patient prior to moving ahead with any transplant in order to choose the best approach. Because the number of pediatric patients undergoing transplantation is relatively small, it is strongly recommended that when available, patients be treated in the context of a clinical trial. Recommendations on whom and when to transplant are frequently being updated, so it is important that transplant physicians and referring physicians carefully review therapeutic options for each patient.

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References

  1. Appelbaum FR, Forman SJ, Negrin RS, Antin JH. Thomas’ hematopoietic cell transplantation stem cell transplantation. Chichester: Wiley; 2015. Available from: https://libproxy.usc.edu/login?url=http://ZB5LH7ED7A.search.serialssolutions.com/?V=1.0&L=ZB5LH7ED7A&S=JCs&C=TC0001420526&T=marc.

  2. Bortin MM. A compendium of reported human bone marrow transplants. Transplantation. 1970;9(6):571–87

    Article  CAS  PubMed  Google Scholar 

  3. Oliansky DM, Camitta B, Gaynon P, Nieder ML, Parsons SK, Pulsipher MA, et al. Role of cytotoxic therapy with hematopoietic stem cell transplantation in the treatment of pediatric acute lymphoblastic leukemia: update of the 2005 evidence-based review. Biol Blood Marrow Transplant. 2012;18(4):505–22

    Article  CAS  PubMed  Google Scholar 

  4. Schrauder A, von Stackelberg A, Schrappe M, Cornish J, Peters C, Group A-BS, et al. Allogeneic hematopoietic SCT in children with ALL: current concepts of ongoing prospective SCT trials. Bone Marrow Transplant. 2008;41(Suppl 2):S71–4

    Article  PubMed  Google Scholar 

  5. Pulsipher MA, Peters C, Pui CH. High-risk pediatric acute lymphoblastic leukemia: to transplant or not to transplant? Biol Blood Marrow Transplant. 2011;17(1 Suppl):S137–48

    Article  PubMed  PubMed Central  Google Scholar 

  6. Nachman JB, Heerema NA, Sather H, Camitta B, Forestier E, Harrison CJ, et al. Outcome of treatment in children with hypodiploid acute lymphoblastic leukemia. Blood. 2007;110(4):1112–5

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  7. Mehta PA, Zhang MJ, Eapen M, He W, Seber A, Gibson B, et al. Transplantation outcomes for children with hypodiploid acute lymphoblastic leukemia. Biol Blood Marrow Transplant. 2015;21(7):1273–7

    Article  PubMed  PubMed Central  Google Scholar 

  8. Mullighan CG, Jeha S, Pei D, Payne-Turner D, Coustan-Smith E, Roberts KG, et al. Outcome of children with hypodiploid ALL treated with risk-directed therapy based on MRD levels. Blood. 2015;126(26):2896–9

    Article  PubMed  PubMed Central  Google Scholar 

  9. Mann G, Attarbaschi A, Schrappe M, De Lorenzo P, Peters C, Hann I, et al. Improved outcome with hematopoietic stem cell transplantation in a poor prognostic subgroup of infants with mixed-lineage-leukemia (MLL)-rearranged acute lymphoblastic leukemia: results from the Interfant-99 study. Blood. 2010;116(15):2644–50

    Article  CAS  PubMed  Google Scholar 

  10. Racadio JM, Reaman GH, Smith FO. Hematopoietic cell transplantation in children with cancer. New York: Springer; 2013. Available from: https://libproxy.usc.edu/login?url=http://link.springer.com/10.1007/978-3-642-39920-6.

  11. Schultz KR, Carroll A, Heerema NA, Bowman WP, Aledo A, Slayton WB, et al. Long-term follow-up of imatinib in pediatric Philadelphia chromosome-positive acute lymphoblastic leukemia: Children’s Oncology Group study AALL0031. Leukemia. 2014;28(7):1467–71

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  12. Biondi A, Schrappe M, De Lorenzo P, Castor A, Lucchini G, Gandemer V, et al. Imatinib after induction for treatment of children and adolescents with Philadelphia-chromosome-positive acute lymphoblastic leukaemia (EsPhALL): a randomised, open-label, intergroup study. Lancet Oncol. 2012;13(9):936–45

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  13. Schrauder A, Reiter A, Gadner H, Niethammer D, Klingebiel T, Kremens B, et al. Superiority of allogeneic hematopoietic stem-cell transplantation compared with chemotherapy alone in high-risk childhood T-cell acute lymphoblastic leukemia: results from ALL-BFM 90 and 95. J Clin Oncol. 2006;24(36):5742–9

    Article  PubMed  Google Scholar 

  14. Conter V, Bartram CR, Valsecchi MG, Schrauder A, Panzer-Grümayer R, Möricke A, et al. Molecular response to treatment redefines all prognostic factors in children and adolescents with B-cell precursor acute lymphoblastic leukemia: results in 3184 patients of the AIEOP-BFM ALL 2000 study. Blood. 2010;115(16):3206–14

    Article  CAS  PubMed  Google Scholar 

  15. Hagedorn N, Acquaviva C, Fronkova E, von Stackelberg A, Barth A, zur Stadt U, et al. Submicroscopic bone marrow involvement in isolated extramedullary relapses in childhood acute lymphoblastic leukemia: a more precise definition of “isolated” and its possible clinical implications, a collaborative study of the Resistant Disease Committee of the International BFM study group. Blood. 2007;110(12):4022–9

    Article  CAS  PubMed  Google Scholar 

  16. Masurekar AN, Parker CA, Shanyinde M, Moorman AV, Hancock JP, Sutton R, et al. Outcome of central nervous system relapses in childhood acute lymphoblastic leukaemia–prospective open cohort analyses of the ALLR3 trial. PLoS One. 2014;9(10):e108107

    Article  PubMed  PubMed Central  CAS  Google Scholar 

  17. Eapen M, Zhang MJ, Devidas M, Raetz E, Barredo JC, Ritchey AK, et al. Outcomes after HLA-matched sibling transplantation or chemotherapy in children with acute lymphoblastic leukemia in a second remission after an isolated central nervous system relapse: a collaborative study of the Children’s Oncology Group and the Center for International Blood and Marrow Transplant Research. Leukemia. 2008;22(2):281–6

    Article  CAS  PubMed  Google Scholar 

  18. Andreola G, Labopin M, Beelen D, Chevallier P, Tabrizi R, Bosi A, et al. Long-term outcome and prognostic factors of second allogeneic hematopoietic stem cell transplant for acute leukemia in patients with a median follow-up of ≥10 years. Bone Marrow Transplant. 2015;50(12):1508–12

    Article  CAS  PubMed  Google Scholar 

  19. Bosi A, Laszlo D, Labopin M, Reffeirs J, Michallet M, Gluckman E, et al. Second allogeneic bone marrow transplantation in acute leukemia: results of a survey by the European Cooperative Group for Blood and Marrow Transplantation. J Clin Oncol. 2001;19(16):3675–84

    Article  CAS  PubMed  Google Scholar 

  20. Shah AJ, Kapoor N, Weinberg KI, Crooks GM, Kohn DB, Lenarsky C, et al. Second hematopoietic stem cell transplantation in pediatric patients: overall survival and long-term follow-up. Biol Blood Marrow Transplant. 2002;8(4):221–8

    Article  PubMed  Google Scholar 

  21. Pulsipher MA, Boucher KM, Wall D, Frangoul H, Duval M, Goyal RK, 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. 2009;114(7):1429–36

    Article  CAS  PubMed  Google Scholar 

  22. Thol F, Schlenk RF, Heuser M, Ganser A. How I treat refractory and early relapsed acute myeloid leukemia. Blood. 2015;126(3):319–27

    Article  CAS  PubMed  Google Scholar 

  23. Rubnitz JE, Razzouk BI, Lensing S, Pounds S, Pui CH, Ribeiro RC. Prognostic factors and outcome of recurrence in childhood acute myeloid leukemia. Cancer. 2007;109(1):157–63

    Article  PubMed  Google Scholar 

  24. Webb DK, Wheatley K, Harrison G, Stevens RF, Hann IM. Outcome for children with relapsed acute myeloid leukaemia following initial therapy in the Medical Research Council (MRC) AML 10 trial. MRC Childhood Leukaemia Working Party. Leukemia. 1999;13(1):25–31

    Article  CAS  PubMed  Google Scholar 

  25. Bunin NJ, Davies SM, Aplenc R, Camitta BM, DeSantes KB, Goyal RK, et al. Unrelated donor bone marrow transplantation for children with acute myeloid leukemia beyond first remission or refractory to chemotherapy. J Clin Oncol. 2008;26(26):4326–32

    Article  PubMed  PubMed Central  Google Scholar 

  26. Godley LA, Larson RA. Therapy-related myeloid leukemia. Semin Oncol. 2008;35(4):418–29

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  27. Levis M. FLT3 mutations in acute myeloid leukemia: what is the best approach in 2013? Hematology Am Soc Hematol Educ Program. 2013;2013:220–6

    PubMed  PubMed Central  Google Scholar 

  28. Meshinchi S, Alonzo TA, Stirewalt DL, Zwaan M, Zimmerman M, Reinhardt D, et al. Clinical implications of FLT3 mutations in pediatric AML. Blood. 2006;108(12):3654–61

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  29. Kuwatsuka Y, Miyamura K, Suzuki R, Kasai M, Maruta A, Ogawa H, et al. Hematopoietic stem cell transplantation for core binding factor acute myeloid leukemia: t(8;21) and inv(16) represent different clinical outcomes. Blood. 2009;113(9):2096–103

    Article  CAS  PubMed  Google Scholar 

  30. Brown P, McIntyre E, Rau R, Meshinchi S, Lacayo N, Dahl G, et al. The incidence and clinical significance of nucleophosmin mutations in childhood AML. Blood. 2007;110(3):979–85

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  31. Ho PA, Alonzo TA, Gerbing RB, Pollard J, Stirewalt DL, Hurwitz C, et al. Prevalence and prognostic implications of CEBPA mutations in pediatric acute myeloid leukemia (AML): a report from the Children’s Oncology Group. Blood. 2009;113(26):6558–66

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  32. de Botton S, Coiteux V, Chevret S, Rayon C, Vilmer E, Sanz M, et al. Outcome of childhood acute promyelocytic leukemia with all-trans-retinoic acid and chemotherapy. J Clin Oncol. 2004;22(8):1404–12

    Article  PubMed  CAS  Google Scholar 

  33. de Botton S, Fawaz A, Chevret S, Dombret H, Thomas X, Sanz M, et al. Autologous and allogeneic stem-cell transplantation as salvage treatment of acute promyelocytic leukemia initially treated with all-trans-retinoic acid: a retrospective analysis of the European Acute Promyelocytic Leukemia Group. J Clin Oncol. 2005;23(1):120–6

    Article  PubMed  CAS  Google Scholar 

  34. Dvorak CC, Agarwal R, Dahl GV, Gregory JJ, Feusner JH. Hematopoietic stem cell transplant for pediatric acute promyelocytic leukemia. Biol Blood Marrow Transplant. 2008;14(7):824–30

    Article  PubMed  PubMed Central  Google Scholar 

  35. Greenberg P, Cox C, LeBeau MM, Fenaux P, Morel P, Sanz G, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997;89(6):2079–88

    CAS  PubMed  Google Scholar 

  36. Vardiman JW, Thiele J, Arber DA, Brunning RD, Borowitz MJ, Porwit A, et al. The 2008 revision of the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia: rationale and important changes. Blood. 2009;114(5):937–51

    Article  CAS  PubMed  Google Scholar 

  37. Oliansky DM, Antin JH, Bennett JM, Deeg HJ, Engelhardt C, Heptinstall KV, et al. The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of myelodysplastic syndromes: an evidence-based review. Biol Blood Marrow Transplant. 2009;15(2):137–72

    Article  PubMed  Google Scholar 

  38. Stone RM. How I treat patients with myelodysplastic syndromes. Blood. 2009;113(25):6296–303

    Article  CAS  PubMed  Google Scholar 

  39. Alessandrino EP, Della Porta MG, Bacigalupo A, Van Lint MT, Falda M, Onida F, et al. WHO classification and WPSS predict posttransplantation outcome in patients with myelodysplastic syndrome: a study from the Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Blood. 2008;112(3):895–902

    Article  CAS  PubMed  Google Scholar 

  40. Pasquet M, Bellanné-Chantelot C, Tavitian S, Prade N, Beaupain B, Larochelle O, et al. High frequency of GATA2 mutations in patients with mild chronic neutropenia evolving to MonoMac syndrome, myelodysplasia, and acute myeloid leukemia. Blood. 2013;121(5):822–9

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  41. Strahm B, Nollke P, Zecca M, Korthof ET, Bierings M, Furlan I, et al. Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study. Leukemia. 2011;25(3):455–62

    Article  CAS  PubMed  Google Scholar 

  42. Locatelli F, Nollke P, Zecca M, Korthof E, Lanino E, Peters C, et al. Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML): results of the EWOG-MDS/EBMT trial. Blood. 2005;105(1):410–9

    Article  CAS  PubMed  Google Scholar 

  43. Loh ML. Recent advances in the pathogenesis and treatment of juvenile myelomonocytic leukaemia. Br J Haematol. 2011;152(6):677–87

    Article  CAS  PubMed  Google Scholar 

  44. Locatelli F, Niemeyer CM. How I treat juvenile myelomonocytic leukemia. Blood. 2015;125(7):1083–90

    Article  CAS  PubMed  Google Scholar 

  45. Bergstraesser E, Hasle H, Rogge T, Fischer A, Zimmermann M, Noellke P, et al. Non-hematopoietic stem cell transplantation treatment of juvenile myelomonocytic leukemia: a retrospective analysis and definition of response criteria. Pediatr Blood Cancer. 2007;49(5):629–33

    Article  PubMed  Google Scholar 

  46. Pulsipher MA. Treatment of CML in pediatric patients: should imatinib mesylate (STI-571, Gleevec) or allogeneic hematopoietic cell transplant be front-line therapy? Pediatr Blood Cancer. 2004;43(5):523–33

    Article  PubMed  Google Scholar 

  47. Hijiya N, Schultz KR, Metzler M, Millot F, Suttorp M. Pediatric chronic myeloid leukemia is a unique disease that requires a different approach. Blood. 2016;127(4):392–9

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  48. Chaudhury S, Sparapani R, Hu ZH, Nishihori T, Abdel-Azim H, Malone A, et al. Outcomes of allogeneic hematopoietic cell transplantation in children (<18y) and young adults (18-29y) with chronic myeloid leukemia: a CIBMTR cohort analysis. Biol Blood Marrow Transplant. 2016;22:1056–64

    Article  PubMed  PubMed Central  Google Scholar 

  49. Perales MA, Ceberio I, Armand P, Burns LJ, Chen R, Cole PD, et al. Role of cytotoxic therapy with hematopoietic cell transplantation in the treatment of Hodgkin lymphoma: guidelines from the American Society for Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2015;21(6):971–83

    Article  PubMed  Google Scholar 

  50. Reddy NM, Perales MA. Stem cell transplantation in Hodgkin lymphoma. Hematol Oncol Clin North Am. 2014;28(6):1097–112

    Article  PubMed  Google Scholar 

  51. von Tresckow B, Moskowitz CH. Treatment of relapsed and refractory Hodgkin lymphoma. Semin Hematol. 2016;53(3):180–5

    Article  Google Scholar 

  52. Rancea M, von Tresckow B, Monsef I, Engert A, Skoetz N. High-dose chemotherapy followed by autologous stem cell transplantation for patients with relapsed or refractory Hodgkin lymphoma: a systematic review with meta-analysis. Crit Rev Oncol Hematol. 2014;92(1):1–10

    Article  PubMed  Google Scholar 

  53. Harker-Murray PD, Drachtman RA, Hodgson DC, Chauvenet AR, Kelly KM, Cole PD. Stratification of treatment intensity in relapsed pediatric Hodgkin lymphoma. Pediatr Blood Cancer. 2014;61(4):579–86

    Article  PubMed  Google Scholar 

  54. Metzger ML, Hudson MM, Krasin MJ, Wu J, Kaste SC, Kun LE, et al. Initial response to salvage therapy determines prognosis in relapsed pediatric Hodgkin lymphoma patients. Cancer. 2010;116(18):4376–84

    Article  PubMed  PubMed Central  Google Scholar 

  55. Moskowitz CH, Matasar MJ, Zelenetz AD, Nimer SD, Gerecitano J, Hamlin P, et al. Normalization of pre-ASCT, FDG-PET imaging with second-line, non-cross-resistant, chemotherapy programs improves event-free survival in patients with Hodgkin lymphoma. Blood. 2012;119(7):1665–70

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  56. Chen R, Gopal AK, Smith SE, Ansell SM, Rosenblatt JD, Savage KJ, et al. Five-year survival and durability results of brentuximab vedotin in patients with relapsed or refractory Hodgkin lymphoma. Blood. 2016;128:1562–6

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  57. Ansell SM, Lesokhin AM, Borrello I, Halwani A, Scott EC, Gutierrez M, et al. PD-1 blockade with nivolumab in relapsed or refractory Hodgkin’s lymphoma. N Engl J Med. 2015;372(4):311–9

    Article  PubMed  CAS  Google Scholar 

  58. Devetten MP, Hari PN, Carreras J, Logan BR, van Besien K, Bredeson CN, et al. Unrelated donor reduced-intensity allogeneic hematopoietic stem cell transplantation for relapsed and refractory Hodgkin lymphoma. Biol Blood Marrow Transplant. 2009;15(1):109–17

    Article  PubMed  PubMed Central  Google Scholar 

  59. Claviez A, Canals C, Dierickx D, Stein J, Badell I, Pession A, et al. Allogeneic hematopoietic stem cell transplantation in children and adolescents with recurrent and refractory Hodgkin lymphoma: an analysis of the European Group for Blood and Marrow Transplantation. Blood. 2009;114(10):2060–7

    Article  CAS  PubMed  Google Scholar 

  60. Smith MA, Altekruse SF, Adamson PC, Reaman GH, Seibel NL. Declining childhood and adolescent cancer mortality. Cancer. 2014;120(16):2497–506

    Article  PubMed  PubMed Central  Google Scholar 

  61. Gross TG, Hale GA, He W, Camitta BM, Sanders JE, Cairo MS, et al. Hematopoietic stem cell transplantation for refractory or recurrent non-Hodgkin lymphoma in children and adolescents. Biol Blood Marrow Transplant. 2010;16(2):223–30

    Article  PubMed  Google Scholar 

  62. Harris RE, Termuhlen AM, Smith LM, Lynch J, Henry MM, Perkins SL, et al. Autologous peripheral blood stem cell transplantation in children with refractory or relapsed lymphoma: results of Children’s Oncology Group study A5962. Biol Blood Marrow Transplant. 2011;17(2):249–58

    Article  PubMed  Google Scholar 

  63. Satwani P, Jin Z, Martin PL, Bhatia M, Garvin JH, George D, et al. Sequential myeloablative autologous stem cell transplantation and reduced intensity allogeneic hematopoietic cell transplantation is safe and feasible in children, adolescents and young adults with poor-risk refractory or recurrent Hodgkin and non-Hodgkin lymphoma. Leukemia. 2015;29(2):448–55

    Article  CAS  PubMed  Google Scholar 

  64. Schuster FR, Stanglmaier M, Woessmann W, Winkler B, Siepermann M, Meisel R, et al. Immunotherapy with the trifunctional anti-CD20 x anti-CD3 antibody FBTA05 (Lymphomun) in paediatric high-risk patients with recurrent CD20-positive B cell malignancies. Br J Haematol. 2015;169(1):90–102

    Article  CAS  PubMed  Google Scholar 

  65. Dunleavy K, Pittaluga S, Maeda LS, Advani R, Chen CC, Hessler J, et al. Dose-adjusted EPOCH-rituximab therapy in primary mediastinal B-cell lymphoma. N Engl J Med. 2013;368(15):1408–16

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  66. Brugières L, Pacquement H, Le Deley MC, Leverger G, Lutz P, Paillard C, et al. Single-drug vinblastine as salvage treatment for refractory or relapsed anaplastic large-cell lymphoma: a report from the French Society of Pediatric Oncology. J Clin Oncol. 2009;27(30):5056–61

    Article  PubMed  CAS  Google Scholar 

  67. Kung FH, Harris MB, Krischer JP. Ifosfamide/carboplatin/etoposide (ICE), an effective salvaging therapy for recurrent malignant non-Hodgkin lymphoma of childhood: a Pediatric Oncology Group phase II study. Med Pediatr Oncol. 1999;32(3):225–6

    Article  CAS  PubMed  Google Scholar 

  68. Strullu M, Thomas C, Le Deley MC, Chevance A, Kanold J, Bertrand Y, et al. Hematopoietic stem cell transplantation in relapsed ALK+ anaplastic large cell lymphoma in children and adolescents: a study on behalf of the SFCE and SFGM-TC. Bone Marrow Transplant. 2015;50(6):795–801

    Article  CAS  PubMed  Google Scholar 

  69. Mossé YP, Lim MS, Voss SD, Wilner K, Ruffner K, Laliberte J, et al. Safety and activity of crizotinib for paediatric patients with refractory solid tumours or anaplastic large-cell lymphoma: a Children’s Oncology Group phase 1 consortium study. Lancet Oncol. 2013;14(6):472–80

    Article  PubMed  PubMed Central  CAS  Google Scholar 

  70. Pro B, Advani R, Brice P, Bartlett NL, Rosenblatt JD, Illidge T, et al. Brentuximab vedotin (SGN-35) in patients with relapsed or refractory systemic anaplastic large-cell lymphoma: results of a phase II study. J Clin Oncol. 2012;30(18):2190–6

    Article  CAS  PubMed  Google Scholar 

  71. Matthay KK, Reynolds CP, Seeger RC, Shimada H, Adkins ES, Haas-Kogan D, et al. Long-term results for children with high-risk neuroblastoma treated on a randomized trial of myeloablative therapy followed by 13-cis-retinoic acid: a Children’s Oncology Group study. J Clin Oncol. 2009;27(7):1007–13

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  72. Cohn SL, Pearson AD, London WB, Monclair T, Ambros PF, Brodeur GM, et al. The International Neuroblastoma Risk Group (INRG) classification system: an INRG Task Force report. J Clin Oncol. 2009;27(2):289–97

    Article  PubMed  PubMed Central  Google Scholar 

  73. Grupp SA, Asgharzadeh S, Yanik GA. Neuroblastoma: issues in transplantation. Biol Blood Marrow Transplant. 2012;18(1 Suppl):S92–100

    Article  PubMed  PubMed Central  Google Scholar 

  74. Pinto NR, Applebaum MA, Volchenboum SL, Matthay KK, London WB, Ambros PF, et al. Advances in risk classification and treatment strategies for neuroblastoma. J Clin Oncol. 2015;33(27):3008–17

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  75. Park JR, Kreissman SG, London WB, Naranjo A, Cohn SL, Hogarty MD, Tenney SC, Haas-Kogan D, Shaw PJ, Geiger JD, Doski JJ, Gorges SW, Khanna G, Voss SD, Maris JM, Grupp SA, Diller L. A phase III randomized clinical trial (RCT) of tandem myeloablative autologous stem cell transplant (ASCT) using peripheral blood stem cell (PBSC) as consolidation therapy for high-risk neuroblastoma (HR-NB): a Children’s Oncology Group (COG) study. J Clin Oncol. 2016 [suppl; abstr LBA3]. In press.

    Google Scholar 

  76. Ikeda H, Iehara T, Tsuchida Y, Kaneko M, Hata J, Naito H, et al. Experience with international neuroblastoma staging system and pathology classification. Br J Cancer. 2002;86(7):1110–6

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  77. Monclair T, Brodeur GM, Ambros PF, Brisse HJ, Cecchetto G, Holmes K, et al. The International Neuroblastoma Risk Group (INRG) staging system: an INRG Task Force report. J Clin Oncol. 2009;27(2):298–303

    Article  PubMed  PubMed Central  Google Scholar 

  78. Saha A, Salley CG, Saigal P, Rolnitzky L, Goldberg J, Scott S, et al. Late effects in survivors of childhood CNS tumors treated on Head Start I and II protocols. Pediatr Blood Cancer. 2014;61(9):1644–52; quiz 53–72.

    Article  PubMed  PubMed Central  Google Scholar 

  79. Mulhern RK, Palmer SL, Merchant TE, Wallace D, Kocak M, Brouwers P, et al. Neurocognitive consequences of risk-adapted therapy for childhood medulloblastoma. J Clin Oncol. 2005;23(24):5511–9

    Article  PubMed  Google Scholar 

  80. Dhall G, Grodman H, Ji L, Sands S, Gardner S, Dunkel IJ, et al. Outcome of children less than three years old at diagnosis with non-metastatic medulloblastoma treated with chemotherapy on the “Head Start” I and II protocols. Pediatr Blood Cancer. 2008;50(6):1169–75

    Article  PubMed  Google Scholar 

  81. Dunkel IJ, Gardner SL, Garvin JH, Goldman S, Shi W, Finlay JL. High-dose carboplatin, thiotepa, and etoposide with autologous stem cell rescue for patients with previously irradiated recurrent medulloblastoma. Neuro Oncol. 2010;12(3):297–303

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  82. Bakst RL, Dunkel IJ, Gilheeney S, Khakoo Y, Becher O, Souweidane MM, et al. Reirradiation for recurrent medulloblastoma. Cancer. 2011;117(21):4977–82

    Article  PubMed  Google Scholar 

  83. Tekautz TM, Fuller CE, Blaney S, Fouladi M, Broniscer A, Merchant TE, et al. Atypical teratoid/rhabdoid tumors (ATRT): improved survival in children 3 years of age and older with radiation therapy and high-dose alkylator-based chemotherapy. J Clin Oncol. 2005;23(7):1491–9

    Article  CAS  PubMed  Google Scholar 

  84. Lafay-Cousin L, Hawkins C, Carret AS, Johnston D, Zelcer S, Wilson B, et al. Central nervous system atypical teratoid rhabdoid tumours: the Canadian Paediatric Brain Tumour Consortium experience. Eur J Cancer. 2012;48(3):353–9

    Article  CAS  PubMed  Google Scholar 

  85. Gardner SL, Asgharzadeh S, Green A, Horn B, McCowage G, Finlay J. Intensive induction chemotherapy followed by high dose chemotherapy with autologous hematopoietic progenitor cell rescue in young children newly diagnosed with central nervous system atypical teratoid rhabdoid tumors. Pediatr Blood Cancer. 2008;51(2):235–40

    Article  PubMed  Google Scholar 

  86. Zaky W, Dhall G, Ji L, Haley K, Allen J, Atlas M, et al. Intensive induction chemotherapy followed by myeloablative chemotherapy with autologous hematopoietic progenitor cell rescue for young children newly-diagnosed with central nervous system atypical teratoid/rhabdoid tumors: the Head Start III experience. Pediatr Blood Cancer. 2014;61(1):95–101

    Article  PubMed  Google Scholar 

  87. Louis DN, Perry A, Reifenberger G, von Deimling A, Figarella-Branger D, Cavenee WK, et al. The 2016 world health organization classification of tumors of the central nervous system: a summary. Acta Neuropathol. 2016;131(6):803–20

    Article  PubMed  Google Scholar 

  88. Fangusaro J, Finlay J, Sposto R, Ji L, Saly M, Zacharoulis S, et al. Intensive chemotherapy followed by consolidative myeloablative chemotherapy with autologous hematopoietic cell rescue (AuHCR) in young children with newly diagnosed supratentorial primitive neuroectodermal tumors (sPNETs): report of the Head Start I and II experience. Pediatr Blood Cancer. 2008;50(2):312–8

    Article  PubMed  Google Scholar 

  89. Koshy M, Rich S, Merchant TE, Mahmood U, Regine WF, Kwok Y. Post-operative radiation improves survival in children younger than 3 years with intracranial ependymoma. J Neurooncol. 2011;105(3):583–90

    Article  PubMed  Google Scholar 

  90. Mason WP, Goldman S, Yates AJ, Boyett J, Li H, Finlay JL. Survival following intensive chemotherapy with bone marrow reconstitution for children with recurrent intracranial ependymoma–a report of the Children’s Cancer Group. J Neurooncol. 1998;37(2):135–43

    Article  CAS  PubMed  Google Scholar 

  91. Zacharoulis S, Levy A, Chi SN, Gardner S, Rosenblum M, Miller DC, et al. Outcome for young children newly diagnosed with ependymoma, treated with intensive induction chemotherapy followed by myeloablative chemotherapy and autologous stem cell rescue. Pediatr Blood Cancer. 2007;49(1):34–40

    Article  PubMed  Google Scholar 

  92. Venkatramani R, Ji L, Lasky J, Haley K, Judkins A, Zhou S, et al. Outcome of infants and young children with newly diagnosed ependymoma treated on the “Head Start” III prospective clinical trial. J Neurooncol. 2013;113(2):285–91

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  93. Motzer RJ, Nichols CJ, Margolin KA, Bacik J, Richardson PG, Vogelzang NJ, et al. Phase III randomized trial of conventional-dose chemotherapy with or without high-dose chemotherapy and autologous hematopoietic stem-cell rescue as first-line treatment for patients with poor-prognosis metastatic germ cell tumors. J Clin Oncol. 2007;25(3):247–56

    Article  CAS  PubMed  Google Scholar 

  94. Rick O, Bokemeyer C, Beyer J, Hartmann JT, Schwella N, Kingreen D, et al. Salvage treatment with paclitaxel, ifosfamide, and cisplatin plus high-dose carboplatin, etoposide, and thiotepa followed by autologous stem-cell rescue in patients with relapsed or refractory germ cell cancer. J Clin Oncol. 2001;19(1):81–8

    Article  CAS  PubMed  Google Scholar 

  95. Modak S, Gardner S, Dunkel IJ, Balmaceda C, Rosenblum MK, Miller DC, et al. Thiotepa-based high-dose chemotherapy with autologous stem-cell rescue in patients with recurrent or progressive CNS germ cell tumors. J Clin Oncol. 2004;22(10):1934–43

    Article  CAS  PubMed  Google Scholar 

  96. Espinoza JC, Haley K, Patel N, Dhall G, Gardner S, Allen J, et al. Outcome of young children with high-grade glioma treated with irradiation-avoiding intensive chemotherapy regimens: final report of the Head Start II and III trials. Pediatr Blood Cancer. 2016;63:1806–13

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  97. Bouffet E, Raquin M, Doz F, Gentet JC, Rodary C, Demeocq F, et al. Radiotherapy followed by high dose busulfan and thiotepa: a prospective assessment of high dose chemotherapy in children with diffuse pontine gliomas. Cancer. 2000;88(3):685–92

    Article  CAS  PubMed  Google Scholar 

  98. Dunkel IJ, Garvin JH, Goldman S, Ettinger LJ, Kaplan AM, Cairo M, et al. High dose chemotherapy with autologous bone marrow rescue for children with diffuse pontine brain stem tumors. Children’s Cancer Group. J Neurooncol. 1998;37(1):67–73

    Article  CAS  PubMed  Google Scholar 

  99. Lee SH, Yoo KH, Sung KW, Kim JY, Cho EJ, Koo HH, et al. Tandem high-dose chemotherapy and autologous stem cell rescue in children with bilateral advanced retinoblastoma. Bone Marrow Transplant. 2008;42(6):385–91

    Article  CAS  PubMed  Google Scholar 

  100. Dome JS, Graf N, Geller JI, Fernandez CV, Mullen EA, Spreafico F, et al. Advances in wilms tumor treatment and biology: progress through international collaboration. J Clin Oncol. 2015;33(27):2999–3007

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  101. Venkatramani R, Shoureshi P, Malvar J, Zhou S, Mascarenhas L. High dose alkylator therapy for extracranial malignant rhabdoid tumors in children. Pediatr Blood Cancer. 2014;61(8):1357–61

    Article  CAS  PubMed  Google Scholar 

  102. Gardner SL, Carreras J, Boudreau C, Camitta BM, Adams RH, Chen AR, et al. Myeloablative therapy with autologous stem cell rescue for patients with Ewing sarcoma. Bone Marrow Transplant. 2008;41(10):867–72

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  103. Cook RJ, Wang Z, Arora M, Lazarus HM, Kasow KA, Champagne MA, et al. Clinical outcomes of patients with desmoplastic small round cell tumor of the peritoneum undergoing autologous HCT: a CIBMTR retrospective analysis. Bone Marrow Transplant. 2012;47(11):1455–8

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  104. Forlenza CJ, Kushner BH, Kernan N, Boulad F, Magnan H, Wexler L, et al. Myeloablative chemotherapy with autologous stem cell transplant for desmoplastic small round cell tumor. Sarcoma. 2015;2015:269197

    Article  PubMed  PubMed Central  CAS  Google Scholar 

  105. Scheinberg P, Young NS. How I treat acquired aplastic anemia. Blood. 2012;120(6):1185–96

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  106. Dufour C, Veys P, Carraro E, Bhatnagar N, Pillon M, Wynn R, et al. Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT. Br J Haematol. 2015;171(4):585–94

    Article  CAS  PubMed  Google Scholar 

  107. Dietz AC, Lucchini G, Samarasinghe S, Pulsipher MA. Evolving hematopoietic stem cell transplantation strategies in severe aplastic anemia. Curr Opin Pediatr. 2016;28(1):3–11

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  108. Myers KC, Davies SM. Hematopoietic stem cell transplantation for bone marrow failure syndromes in children. Biol Blood Marrow Transplant. 2009;15(3):279–92

    Article  PubMed  Google Scholar 

  109. Choi SW, Levine J. Indications for hematopoietic cell transplantation for children with severe congenital neutropenia. Pediatr Transplant. 2010;14(8):937–9

    Article  PubMed  Google Scholar 

  110. Shimamura A, Alter BP. Pathophysiology and management of inherited bone marrow failure syndromes. Blood Rev. 2010;24(3):101–22

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  111. Matos-Fernandez NA, Abou Mourad YR, Caceres W, Kharfan-Dabaja MA. Current status of allogeneic hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria. Biol Blood Marrow Transplant. 2009;15(6):656–61

    Article  CAS  PubMed  Google Scholar 

  112. Ayas M, Nassar A, Hamidieh AA, Kharfan-Dabaja M, Othman TB, Elhaddad A, et al. Reduced intensity conditioning is effective for hematopoietic SCT in dyskeratosis congenita-related BM failure. Bone Marrow Transplant. 2013;48(9):1168–72

    Article  CAS  PubMed  Google Scholar 

  113. Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Pai SY, et al. Primary Immune Deficiency Treatment Consortium (PIDTC) report. J Allergy Clin Immunol. 2014;133(2):335–47

    Article  PubMed  Google Scholar 

  114. Buckley RH. Molecular defects in human severe combined immunodeficiency and approaches to immune reconstitution. Annu Rev Immunol. 2004;22:625–55

    Article  CAS  PubMed  Google Scholar 

  115. Albuquerque W, Gaspar HB. Bilateral sensorineural deafness in adenosine deaminase-deficient severe combined immunodeficiency. J Pediatr. 2004;144(2):278–80

    Article  PubMed  Google Scholar 

  116. Corneo B, Moshous D, Güngör T, Wulffraat N, Philippet P, Le Deist FL, et al. Identical mutations in RAG1 or RAG2 genes leading to defective V(D)J recombinase activity can cause either T-B-severe combined immune deficiency or Omenn syndrome. Blood. 2001;97(9):2772–6

    Article  CAS  PubMed  Google Scholar 

  117. Brown L, Xu-Bayford J, Allwood Z, Slatter M, Cant A, Davies EG, et al. Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening. Blood. 2011;117(11):3243–6

    Article  CAS  PubMed  Google Scholar 

  118. Myers LA, Patel DD, Puck JM, Buckley RH. Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival. Blood. 2002;99(3):872–8

    Article  CAS  PubMed  Google Scholar 

  119. Gaspar HB, Qasim W, Davies EG, Rao K, Amrolia PJ, Veys P. How I treat severe combined immunodeficiency. Blood. 2013;122(23):3749–58

    Article  CAS  PubMed  Google Scholar 

  120. Moratto D, Giliani S, Bonfim C, Mazzolari E, Fischer A, Ochs HD, et al. Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study. Blood. 2011;118(6):1675–84

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  121. Eapen M, DeLaat CA, Baker KS, Cairo MS, Cowan MJ, Kurtzberg J, et al. Hematopoietic cell transplantation for Chediak-Higashi syndrome. Bone Marrow Transplant. 2007;39(7):411–5

    Article  CAS  PubMed  Google Scholar 

  122. Güngör T, Teira P, Slatter M, Stussi G, Stepensky P, Moshous D, et al. Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. Lancet. 2014;383(9915):436–48

    Article  PubMed  CAS  Google Scholar 

  123. Mehta B, Mahadeo K, Kapoor N, Abdel-Azim H. Low-dose total-body irradiation and alemtuzumab-based reduced-intensity conditioning regimen results in durable engraftment and correction of clinical disease among children with chronic granulomatous disease. Pediatr Transplant. 2015;19(4):408–12

    Article  CAS  PubMed  Google Scholar 

  124. Tewari P, Martin PL, Mendizabal A, Parikh SH, Page KM, Driscoll TA, et al. Myeloablative transplantation using either cord blood or bone marrow leads to immune recovery, high long-term donor chimerism and excellent survival in chronic granulomatous disease. Biol Blood Marrow Transplant. 2012;18(9):1368–77

    Article  PubMed  PubMed Central  Google Scholar 

  125. Jordan MB, Allen CE, Weitzman S, Filipovich AH, McClain KL. How I treat hemophagocytic lymphohistiocytosis. Blood. 2011;118(15):4041–52

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  126. Trottestam H, Horne A, Aricò M, Egeler RM, Filipovich AH, Gadner H, et al. Chemoimmunotherapy for hemophagocytic lymphohistiocytosis: long-term results of the HLH-94 treatment protocol. Blood. 2011;118(17):4577–84

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  127. King AA, Kamani N, Bunin N, Sahdev I, Brochstein J, Hayashi RJ, et al. Successful matched sibling donor marrow transplantation following reduced intensity conditioning in children with hemoglobinopathies. Am J Hematol. 2015;90(12):1093–8

    Article  CAS  PubMed  Google Scholar 

  128. Angelucci E, Baronciani D. Allogeneic stem cell transplantation for thalassemia major. Haematologica. 2008;93(12):1780–4

    Article  PubMed  Google Scholar 

  129. Angelucci E, Matthes-Martin S, Baronciani D, Bernaudin F, Bonanomi S, Cappellini MD, et al. Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel. Haematologica. 2014;99(5):811–20

    Article  PubMed  PubMed Central  Google Scholar 

  130. Lucarelli G, Galimberti M, Polchi P, Angelucci E, Baronciani D, Giardini C, et al. Marrow transplantation in patients with thalassemia responsive to iron chelation therapy. N Engl J Med. 1993;329(12):840–4

    Article  CAS  PubMed  Google Scholar 

  131. Panepinto JA, Walters MC, Carreras J, Marsh J, Bredeson CN, Gale RP, et al. Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research. Br J Haematol. 2007;137(5):479–85

    Article  PubMed  Google Scholar 

  132. Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J, et al. 1994 consensus conference on acute GVHD grading. Bone Marrow Transplant. 1995;15(6):825–8

    CAS  PubMed  Google Scholar 

  133. Locatelli F, Kabbara N, Ruggeri A, Ghavamzadeh A, Roberts I, Li CK, et al. Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling. Blood. 2013;122(6):1072–8

    Article  CAS  PubMed  Google Scholar 

  134. Dedeken L, Lê PQ, Azzi N, Brachet C, Heijmans C, Huybrechts S, et al. Haematopoietic stem cell transplantation for severe sickle cell disease in childhood: a single centre experience of 50 patients. Br J Haematol. 2014;165(3):402–8

    Article  CAS  PubMed  Google Scholar 

  135. Maheshwari S, Kassim A, Yeh RF, Domm J, Calder C, Evans M, et al. Targeted Busulfan therapy with a steady-state concentration of 600-700 ng/mL in patients with sickle cell disease receiving HLA-identical sibling bone marrow transplant. Bone Marrow Transplant. 2014;49(3):366–9

    Article  CAS  PubMed  Google Scholar 

  136. Lucarelli G, Isgrò A, Sodani P, Marziali M, Gaziev J, Paciaroni K, et al. Hematopoietic SCT for the Black African and non-Black African variants of sickle cell anemia. Bone Marrow Transplant. 2014;49(11):1376–81

    Article  CAS  PubMed  Google Scholar 

  137. Shenoy S. Hematopoietic stem-cell transplantation for sickle cell disease: current evidence and opinions. Ther Adv Hematol. 2013;4(5):335–44

    Article  PubMed  PubMed Central  Google Scholar 

  138. Kamani NR, Walters MC, Carter S, Aquino V, Brochstein JA, Chaudhury 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. 2012;18(8):1265–72

    Article  PubMed  PubMed Central  Google Scholar 

  139. Bolaños-Meade J, Fuchs EJ, Luznik L, Lanzkron SM, Gamper CJ, Jones RJ, et al. HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease. Blood. 2012;120(22):4285–91

    Article  PubMed  PubMed Central  CAS  Google Scholar 

  140. Dallas MH, Triplett B, Shook DR, Hartford C, Srinivasan A, Laver J, et al. Long-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2013;19(5):820–30

    Article  PubMed  PubMed Central  Google Scholar 

  141. Wynn R. Stem cell transplantation in inherited metabolic disorders. Hematology Am Soc Hematol Educ Program. 2011;2011:285–91

    PubMed  Google Scholar 

  142. Boelens JJ, Prasad VK, Tolar J, Wynn RF, Peters C. Current international perspectives on hematopoietic stem cell transplantation for inherited metabolic disorders. Pediatr Clin North Am. 2010;57(1):123–45

    Article  PubMed  Google Scholar 

  143. Staba SL, Escolar ML, Poe M, Kim Y, Martin PL, Szabolcs P, et al. Cord-blood transplants from unrelated donors in patients with Hurler’s syndrome. N Engl J Med. 2004;350(19):1960–9

    Article  CAS  PubMed  Google Scholar 

  144. Peters C, Charnas LR, Tan Y, Ziegler RS, Shapiro EG, DeFor T, et al. Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999. Blood. 2004;104(3):881–8

    Article  CAS  PubMed  Google Scholar 

  145. Boucher AA, Miller W, Shanley R, Ziegler R, Lund T, Raymond G, et al. Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report. Orphanet J Rare Dis. 2015;10:94

    Article  PubMed  PubMed Central  Google Scholar 

  146. Escolar ML, Poe MD, Provenzale JM, Richards KC, Allison J, Wood S, et al. Transplantation of umbilical-cord blood in babies with infantile Krabbe’s disease. N Engl J Med. 2005;352(20):2069–81

    Article  CAS  PubMed  Google Scholar 

  147. Prasad VK, Mendizabal A, Parikh SH, Szabolcs P, Driscoll TA, Page K, et al. Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes. Blood. 2008;112(7):2979–89

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  148. Boelens JJ. Trends in haematopoietic cell transplantation for inborn errors of metabolism. J Inherit Metab Dis. 2006;29(2-3):413–20

    Article  PubMed  Google Scholar 

  149. Miller WP, Rothman SM, Nascene D, Kivisto T, DeFor TE, Ziegler RS, et al. Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report. Blood. 2011;118(7):1971–8

    Article  CAS  PubMed  Google Scholar 

  150. Peters C. Hematopoietic cell transplantation for storage diseases. Thomas’ hematopoietic cell transplantation: Wiley-Blackwell; 2009. p. 1136–1162.

    Google Scholar 

  151. van Rappard DF, Boelens JJ, Wolf NI. Metachromatic leukodystrophy: disease spectrum and approaches for treatment. Best Pract Res Clin Endocrinol Metab. 2015;29(2):261–73

    Article  PubMed  Google Scholar 

  152. Orchard PJ, Fasth AL, Le Rademacher J, He W, Boelens JJ, Horwitz EM, et al. Hematopoietic stem cell transplantation for infantile osteopetrosis. Blood. 2015;126(2):270–6

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  153. Ott CE, Fischer B, Schröter P, Richter R, Gupta N, Verma N, et al. Severe neuronopathic autosomal recessive osteopetrosis due to homozygous deletions affecting OSTM1. Bone. 2013;55(2):292–7

    Article  CAS  PubMed  Google Scholar 

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    Alicia McFarren & Michael A. Pulsipher

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    Valerie I. Brown

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McFarren, A., Pulsipher, M.A. (2018). Pretransplantation: Indications and Timing. In: Brown, V. (eds) Hematopoietic Stem Cell Transplantation for the Pediatric Hematologist/Oncologist. Springer, Cham. https://doi.org/10.1007/978-3-319-63146-2_4

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