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A prospective study of allogeneic transplantation from unrelated donors for chronic granulomatous disease with target busulfan-based reduced-intensity conditioning

Bone Marrow Transplantation volume 54, pages 168–172 (2019)Cite this article

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To the Editor:

Chronic granulomatous disease (CGD) is a primary immunodisorder caused by impairment of the phagocyte NADPH-oxidase function. Despite improvements in treatment, cases of CGD have been associated with serious infection, episodes of surgery, and hospitalization [1]. Survival probability of CGD patients has improved over the decades, but infections are still a major cause of morbidity and mortality when treated conservatively [2].

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References

  1. Winkelstein JA, Marino MC, Johnston RB Jr., Boyle J, Curnutte J, Gallin JI, et al. Chronic granulomatous disease. Report on a national registry of 368 patients. Medicine (Baltimore). 2000;79:155–69.

    Article  CAS  Google Scholar 

  2. Cole T, Pearce MS, Cant AJ, Cale CM, Goldblatt D, Gennery AR. Clinical outcome in children with chronic granulomatous disease managed conservatively or with hematopoietic stem cell transplantation. J Allergy Clin Immunol. 2013;132:1150–5. https://doi.org/10.1016/j.jaci.2013.05.031.

    Article  PubMed  Google Scholar 

  3. Segal BH, Veys P, Malech H, Cowan MJ. Chronic granulomatous disease: lessons from a rare disorder. Biol Blood Marrow Transplant. 2011;17(1 Suppl):S123–131. https://doi.org/10.1016/j.bbmt.2010.09.008

    Article  CAS  Google Scholar 

  4. Dunogue B, Pilmis B, Mahlaoui N, Elie C, Coignard-Biehler H, Amazzough K, et al. Chronic granulomatous disease in patients reaching adulthood: a nationwide study in France. Clin Infect Dis. 2017;64:767–75. https://doi.org/10.1093/cid/ciw837

    Article  Google Scholar 

  5. 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;18:1368–77. https://doi.org/10.1016/j.bbmt.2012.02.002

    Article  PubMed  PubMed Central  Google Scholar 

  6. Horwitz ME, Barrett AJ, Brown MR, Carter CS, Childs R, Gallin JI, et al. Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft. N Engl J Med. 2001;344:881–8. https://doi.org/10.1056/NEJM200103223441203

    Article  CAS  PubMed  Google Scholar 

  7. Marciano BE, Zerbe CS, Falcone EL, Ding L, DeRavin SS, Daub J, et al. X-linked carriers of chronic granulomatous disease: illness, lyonization, and stability. J Allergy Clin Immunol. 2018;141:365–71. https://doi.org/10.1016/j.jaci.2017.04.035

    Article  CAS  PubMed  Google Scholar 

  8. Gungor 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;383:436–48. https://doi.org/10.1016/S0140-6736(13)62069-3

    Article  CAS  PubMed  Google Scholar 

  9. Matsumoto K, Uchida N, Sakurai A, Taniguchi S, Morita K. Clinical application of the dried blood spot method in the measurement of blood busulfan concentration. Biol Blood Marrow Transplant. 2016;22:1968–73. https://doi.org/10.1016/j.bbmt.2016.07.008

    Article  CAS  PubMed  Google Scholar 

  10. Seger RA. Modern management of chronic granulomatous disease. Br J Haematol. 2008;140:255–66. https://doi.org/10.1111/j.1365-2141.2007.06880.x

    Article  CAS  PubMed  Google Scholar 

  11. Oshrine B, Morsheimer M, Heimall J, Bunin N. Reduced-intensity conditioning for hematopoietic cell transplantation of chronic granulomatous disease. Pediatr Blood Cancer. 2015;62:359–61. https://doi.org/10.1002/pbc.25225

    Article  PubMed  Google Scholar 

  12. Jacobsohn DA, Duerst R, Tse W, Kletzel M. Reduced intensity haemopoietic stem-cell transplantation for treatment of non-malignant diseases in children. Lancet. 2004;364:156–62. https://doi.org/10.1016/S0140-6736(04)16628-2

    Article  PubMed  Google Scholar 

  13. Morillo-Gutierrez B, Beier R, Rao K, Burroughs L, Schulz A, Ewins AM, et al. Treosulfan-based conditioning for allogeneic HSCT in children with chronic granulomatous disease: a multicenter experience. Blood. 2016;128:440–8. https://doi.org/10.1182/blood-2016-03-704015

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  14. Xavier E, Cornillon J, Ruggeri A, Chevallier P, Cornelissen JJ, Andersen NS, et al. Outcomes of cord blood transplantation using reduced-intensity conditioning for chronic lymphocytic leukemia: a Study on Behalf of Eurocord and Cord Blood Committee of Cellular Therapy and Immunobiology Working Party, Chronic Malignancies Working Party of the European Society for Blood and Marrow Transplantation, and the Societe Francaise de Greffe de Moelle et Therapie Cellulaire. Biol Blood Marrow Transplant. 2015;21:1515–23. https://doi.org/10.1016/j.bbmt.2015.04.026

    Article  PubMed  Google Scholar 

  15. DeZern AE, Zahurak M, Symons H, Cooke K, Jones RJ, Brodsky RA. Alternative donor transplantation with high-dose post-transplant cyclophosphamide for refractory severe aplastic anemia. Biol Blood Marrow Transplant. 2016. https://doi.org/10.1016/j.bbmt.2016.12.628

    Article  PubMed  PubMed Central  Google Scholar 

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Acknowledgements

The authors would like to thank Ms. Ayako Okamoto for data management, Prof. Kunihiko Morita for pharmacokinetics of BU, and Dr. Yuji Yamada, Dr. Kaoru Yoshida, Dr. Yui Kimura, Dr. Satoshi Yoshimura, and Dr. Satoshi Miyamoto for patient management. The authors thank a medical editor from the Department of Education for Clinical Research of the National Center for Child Health and Development for assistance in editing this manuscript.

Author contributions

M. Kato is the principal investigator and takes primary responsibility for the paper, designed this study, interpreted data, wrote the manuscript, and gave final approval. T.O. interpreted data and wrote the manuscript. D.T., T.K., E.T., M.T., R.S., M.Y., R.A., Y.T., H.F., Y.S., C.K., K.T., M.O., and K.M. contributed patients and provided clinical data. T.U. and K.I. collected laboratory data. M.S. and E.I. designed this study, interpreted data, and wrote the manuscript. T.T. managed the data. K. Morita calculated and recalculated all busulfan kinetics and cumulative area under the curve. All authors discussed the results and critically reviewed the manuscript. No author has a conflict of interest to disclose.

Funding

This study was supported by a grant from the National Center for Child Health and Development (grant number 28-5 and 29-8), and a grant from the Research Foundation for Clinical Pharmacology.

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Authors and Affiliations

  1. Children’s Cancer Center, National Center for Child Health and Development, Tokyo, Japan

    Tomoo Osumi, Daisuke Tomizawa, Maki Taniguchi, Ryota Shirai, Masanori Yoshida, Rie Ando, Yusuke Tsumura, Yoko Shioda, Chikako Kiyotani, Keita Terashima, Kimikazu Matsumoto & Motohiro Kato

  2. Department of Pediatric Hematology and Oncology Research, National Research Institute for Child Health and Development, Tokyo, Japan

    Tomoo Osumi & Motohiro Kato

  3. Department of Immunology, National Center for Child Health and Development, Tokyo, Japan

    Toshinao Kawai, Eiichiro Tamura & Masafumi Onodera

  4. Department of Clinical Research Promotion, Division of Clinical Trials, Clinical Research Center, National Center for Child Health and Development, Tokyo, Japan

    Mayumi Sako

  5. Medical Informatics, St. Marianna University School of Medicine, Kanagawa, Japan

    Eisuke Inoue

  6. Division of Registration Management, National Center for Child Health and Development, Tokyo, Japan

    Tetsuya Takimoto

  7. Department of Human Genetics, National Research Institute for Child Health and Development, Tokyo, Japan

    Toru Uchiyama & Masafumi Onodera

  8. Department of Advanced Medicine for Infections, National Center for Child Health and Development, Tokyo, Japan

    Ken-Ichi Imadome

  9. Department of Radiology, National Center for Child Health and Development, Tokyo, Japan

    Hiroshi Fuji

  10. Department of Clinical Pharmaceutics, Faculty of Pharmaceutical Sciences, Doshisha Women’s College of Liberal Arts, Kyoto, Japan

    Kana Matsumoto

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  1. Tomoo Osumi
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Correspondence to Motohiro Kato.

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Osumi, T., Tomizawa, D., Kawai, T. et al. A prospective study of allogeneic transplantation from unrelated donors for chronic granulomatous disease with target busulfan-based reduced-intensity conditioning. Bone Marrow Transplant 54, 168–172 (2019). https://doi.org/10.1038/s41409-018-0271-9

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  • DOI: https://doi.org/10.1038/s41409-018-0271-9

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