| Autor: |
Nishimura, Akira, Aoki, Yuki, Ishiwata, Yasuyoshi, Ichimura, Takuya, Ueyama, Junichi, Kawahara, Yuta, Tomoda, Takahiro, Inoue, Maiko, Matsumoto, Kazuaki, Inoue, Kento, Hiroki, Haruka, Ono, Shintaro, Yamashita, Motoi, Okano, Tsubasa, Tanaka-Kubota, Mari, Ashiarai, Miho, Miyamoto, Satoshi, Miyawaki, Reiji, Yamagishi, Chika, Tezuka, Mari |
| Předmět: |
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| Zdroj: |
Journal of Clinical Immunology; Jul2021, Vol. 41 Issue 5, p944-957, 14p |
| Abstrakt: |
Purpose: The purpose of our study was to compare the safety and efficacy of hematopoietic cell transplantation (HCT) using fludarabine (Flu)-based reduced intensity conditioning (RIC) with busulfan (BU) or melphalan (Mel) for primary immunodeficiency diseases (PID). Methods: We retrospectively analyzed transplant outcome, including engraftment, chimerism, immune reconstitution, and complications in 15 patients with severe combined immunodeficiency (SCID) and 27 patients with non-SCID PID. The patients underwent Flu-based RIC-HCT with BU (FluBU: 7 SCID, 16 non-SCID) or Mel (FluMel: 8 SCID, 11 non-SCID). The targeted low-dose BU with therapeutic drug monitoring was set to 30 mg hour/L for SCID. Results: The 2-year overall survival of all patients was 79.6% and that of patients with SCID in the FluBU and FluMel groups was 100% and 62.5%, respectively. In the FluBU group, all seven patients achieved engraftment, good immune reconstitution, and long-term survival. All five patients receiving umbilical cord blood transplantation achieved complete or high-level mixed chimerism and sufficient specific IgG production. In the FluMel group, six of eight patients achieved complete or high-level mixed chimerism. Viral reactivation or new viral infection occurred in one FluBU group patient and four FluMel group patients. In the non-SCID group, 10 of 11 patients (91%) who received FluMel achieved complete or high-level mixed chimerism but had variable outcomes. Patients with WAS (2/2 patients), NEMO deficiency (2/2 patients), and X-linked hyper IgM syndrome (2/3 patients) who received FluBU achieved complete or high-level mixed chimerism and long-term survival. Conclusions: RIC-HCT with FluBU is a safe and effective strategy for obtaining high-level donor chimerism, immune reconstitution including B cell function, and long-term survival in patients with SCID. In patients with non-SCID PID, the results varied according to the subtype of the disease. Further prospective studies are required to optimize the conditioning regimen for non-SCID PID. [ABSTRACT FROM AUTHOR] |
| Databáze: |
Complementary Index |
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