Long-term outcome and chimerism in patients with Wiskott–Aldrich syndrome treated by hematopoietic cell transplantation

a retrospective nationwide survey

Hereditary disorder Working Group of the Japan Society for Hematopoietic Cell Transplantation

Research output: Contribution to journalArticle

Abstract

We analyzed the outcomes of allogeneic stem cell transplantation (SCT) and risk factors for chimerism in 108 patients with Wiskott–Aldrich syndrome (WAS) who were registered with The Japan Society for Hematopoietic Cell Transplantation between January 1985 and December 2016. A preparative conditioning regimen consisting of myeloablative conditioning (MAC) was provided to 76 patients, and reduced-intensity conditioning was provided to 30 patients. Fifty-one patients received prophylaxis against graft-versus-host disease (GVHD) with cyclosporine, and 51 patients received tacrolimus (Tac). Chimerism analyses had been performed in 91 patients. Neutrophil engraftment was achieved in 91 patients (84.3%). The engraftment rate was significantly higher in patients who received Tac for GVHD prophylaxis (p = 0.028). Overall survival rate (OS) was significantly higher in patients with complete chimerism than in patients with mixed chimerism (88.2 ± 6.1% and 66.7 ± 9.9%, respectively, p = 0.003). Multivariate analysis showed that the rate of complete chimerism in patients who received MAC including cyclophosphamide (CY) at a dose of 200 mg/kg was significantly higher (p = 0.021) than that in patients who received other conditioning. Thus, MAC including CY at a dose of 200 mg/kg and Tac for GVHD prophylaxis were optimal conditions of SCT for patients with WAS under existing study.

Original languageEnglish
Pages (from-to)364-369
Number of pages6
JournalInternational journal of hematology
Volume110
Issue number3
DOIs
Publication statusPublished - Sep 20 2019

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Chimerism
Cell Transplantation
Tacrolimus
Graft vs Host Disease
Stem Cell Transplantation
Surveys and Questionnaires
Cyclophosphamide
Stem Cell Factor
Cyclosporine
Japan
Neutrophils

All Science Journal Classification (ASJC) codes

  • Hematology

Cite this

Long-term outcome and chimerism in patients with Wiskott–Aldrich syndrome treated by hematopoietic cell transplantation : a retrospective nationwide survey. / Hereditary disorder Working Group of the Japan Society for Hematopoietic Cell Transplantation.

In: International journal of hematology, Vol. 110, No. 3, 20.09.2019, p. 364-369.

Research output: Contribution to journalArticle

Hereditary disorder Working Group of the Japan Society for Hematopoietic Cell Transplantation. / Long-term outcome and chimerism in patients with Wiskott–Aldrich syndrome treated by hematopoietic cell transplantation : a retrospective nationwide survey. In: International journal of hematology. 2019 ; Vol. 110, No. 3. pp. 364-369.
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abstract = "We analyzed the outcomes of allogeneic stem cell transplantation (SCT) and risk factors for chimerism in 108 patients with Wiskott–Aldrich syndrome (WAS) who were registered with The Japan Society for Hematopoietic Cell Transplantation between January 1985 and December 2016. A preparative conditioning regimen consisting of myeloablative conditioning (MAC) was provided to 76 patients, and reduced-intensity conditioning was provided to 30 patients. Fifty-one patients received prophylaxis against graft-versus-host disease (GVHD) with cyclosporine, and 51 patients received tacrolimus (Tac). Chimerism analyses had been performed in 91 patients. Neutrophil engraftment was achieved in 91 patients (84.3{\%}). The engraftment rate was significantly higher in patients who received Tac for GVHD prophylaxis (p = 0.028). Overall survival rate (OS) was significantly higher in patients with complete chimerism than in patients with mixed chimerism (88.2 ± 6.1{\%} and 66.7 ± 9.9{\%}, respectively, p = 0.003). Multivariate analysis showed that the rate of complete chimerism in patients who received MAC including cyclophosphamide (CY) at a dose of 200 mg/kg was significantly higher (p = 0.021) than that in patients who received other conditioning. Thus, MAC including CY at a dose of 200 mg/kg and Tac for GVHD prophylaxis were optimal conditions of SCT for patients with WAS under existing study.",
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AU - Iguchi, Akihiro

AU - Cho, Yuko

AU - Yabe, Hiromasa

AU - Kato, Shunichi

AU - Kato, Koji

AU - Kato, Koji

AU - Koh, Katsuyoshi

AU - Takita, Junko

AU - Ishihara, Takashi

AU - Inoue, Masami

AU - Imai, Kohsuke

AU - Nakayama, Hideki

AU - Hashii, Yoshiko

AU - Morimoto, Akira

AU - Atsuta, Yoshiko

AU - Morio, Tomohiro

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