Prospective Study of Allogeneic Hematopoietic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide and Antithymocyte Globulin from HLA-Mismatched Related Donors for Nonmalignant Diseases

• Published in: Biology of blood and marrow transplantation 2020-11, Vol.26 (11), p.e286-e291
• Main Authors: Osumi, Tomoo, Yoshimura, Satoshi, Sako, Mayumi, Uchiyama, Toru, Ishikawa, Takashi, Kawai, Toshinao, Inoue, Eisuke, Takimoto, Tetsuya, Takeuchi, Ichiro, Yamada, Masaki, Sakamoto, Kenichi, Yoshida, Kaoru, Kimura, Yui, Matsukawa, Yukihiro, Matsumoto, Kana, Imadome, Ken-Ichi, Arai, Katsuhiro, Deguchi, Takao, Imai, Kohsuke, Yuza, Yuki, Matsumoto, Kimikazu, Onodera, Masafumi, Kanegane, Hirokazu, Tomizawa, Daisuke, Kato, Motohiro
• Format: Article
• Language: English
• Subjects: Graft-versus-host disease; Haploidentical donor; Nonmalignant disease; Post-transplantation cyclophosphamide; Prospective trial
• ISSN: 1083-8791; 1523-6536
• DOI: 10.1016/j.bbmt.2020.08.008

•We conducted a prospective trial of haploidentical hematopoietic stem cell transplantation for nonmalignant diseases.•Post-transplantation cyclophosphamide and low-dose antithymocyte globulin were used as graft-versus-host disease (GVHD) prophylaxis.•All 6 patients achieved engraftment, and none developed severe GVHD.•All patients had sustained full donor chimerism without chronic GVHD. Allogeneic hematopoietic stem cell transplantation (HSCT) is performed as a curative treatment for children with nonmalignant diseases, such as bone marrow failure syndromes and primary immunodeficiencies. Because graft-versus-host-disease (GVHD) is a major factor affecting survival probability and quality of life after HSCT, the availability of HLA-matched donors restricts the application of HSCT. Recently, HSCT with post-transplantation cyclophosphamide (PTCy) has emerged as a potent method to prevent GVHD after HSCT from HLA-haploidentical donors, and some studies have suggested the safety of PTCy-HSCT for nonmalignant diseases. We conducted a prospective clinical trial aiming to help confirm the safety of HSCT and further reduction of GVHD using a combination of PTCy and low-dose antithymocyte globulin (ATG) from HLA-mismatched related donors for children with nonmalignant diseases. Six patients underwent HSCT and achieved engraftment at a median of 14.5 days, and no patient developed severe acute GVHD. All patients had sustained donor chimerism without developing chronic GVHD at the last follow-up. In conclusion, HSCT with PTCy and low-dose ATG from an HLA-mismatched related donor were feasible to control GVHD for nonmalignant diseases in the children involved in our study. © 2020 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc.