Hematopoietic stem cell transplantation for familial hemophagocytic lymphohistiocytosis and Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis in Japan

Shouichi Ohga, Kazuko Kudo, Eiichi Ishii, Satoshi Honjo, Akira Morimoto, Yuko Osugi, Akihisa Sawada, Masami Inoue, Ken Tabuchi, Nobuhiro Suzuki, Yasushi Ishida, Shinsaku Imashuku, Shunichi Kato, Toshiro Hara

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Abstract

Background. Post-transplant outcomes of hemophagocytic lymphohistiocytosis (HLH) patients were analyzed in Japan where Epstein-Barr virus (EBV)-associated severe forms are problematic. Methods. Fifty-seven patients (43 familial HLH [12 FHL2, 11 FHL3, 20 undefined], 14 EBV-HLH) who underwent stem cell transplantation (SCT) between 1995 and 2005 were enrolled based on the nationwide registration. Results. Fifty-seven patients underwent 61 SCTs, including 4 consecutive SCTs. SCTs were employed using allogeneic donors in 93% of cases (allo 53, twin 1, auto 3). Unrelated donor cord blood transplantation (UCBT) was employed in half of cases (21 FHL, 7 EBV-HLH). Reduced intensity conditioning was used in 26% of cases. The 10-year overall survival rates (median±SE%) were 65.0±7.9% in FHL and 85.7±9.4% in EBV-HLH patients, respectively. The survival of UCBT recipients was >65% in both FHL and EBV-HLH patients. Three out of four patients were alive with successful engraftment after second UCBT. FHL patients showed a poorer outcome due to early treatmentrelated deaths (<100 days, seven patients) and a higher incidence of sequelae than EBV-HLH patients (P=0.02). The risk of death for FHL patients having received an unrelated donor bone marrow transplant was marginally higher than that for a related donor SCT (P=0.05) and that for UCBT (P=0.07). Conclusions. EBV-HLH patients had a better prognosis after SCT than FHL patients. FHL patients showed either an equal or better outcome even after UCBT compared with the recent reports. UCB might therefore be acceptable as an alternate SCT source for HLH patients, although the optimal conditioning remains to be determined.

Original languageEnglish
Pages (from-to)299-306
Number of pages8
JournalPediatric Blood and Cancer
Volume54
Issue number2
DOIs
Publication statusPublished - Feb 1 2010

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Hemophagocytic Lymphohistiocytosis
Hematopoietic Stem Cell Transplantation
Human Herpesvirus 4
Japan
Unrelated Donors
Fetal Blood
Stem Cell Transplantation
Transplantation
Tissue Donors
Transplants

All Science Journal Classification (ASJC) codes

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

Cite this

Hematopoietic stem cell transplantation for familial hemophagocytic lymphohistiocytosis and Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis in Japan. / Ohga, Shouichi; Kudo, Kazuko; Ishii, Eiichi; Honjo, Satoshi; Morimoto, Akira; Osugi, Yuko; Sawada, Akihisa; Inoue, Masami; Tabuchi, Ken; Suzuki, Nobuhiro; Ishida, Yasushi; Imashuku, Shinsaku; Kato, Shunichi; Hara, Toshiro.

In: Pediatric Blood and Cancer, Vol. 54, No. 2, 01.02.2010, p. 299-306.

Research output: Contribution to journalArticle

Ohga, S, Kudo, K, Ishii, E, Honjo, S, Morimoto, A, Osugi, Y, Sawada, A, Inoue, M, Tabuchi, K, Suzuki, N, Ishida, Y, Imashuku, S, Kato, S & Hara, T 2010, 'Hematopoietic stem cell transplantation for familial hemophagocytic lymphohistiocytosis and Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis in Japan', Pediatric Blood and Cancer, vol. 54, no. 2, pp. 299-306. https://doi.org/10.1002/pbc.22310
Ohga, Shouichi ; Kudo, Kazuko ; Ishii, Eiichi ; Honjo, Satoshi ; Morimoto, Akira ; Osugi, Yuko ; Sawada, Akihisa ; Inoue, Masami ; Tabuchi, Ken ; Suzuki, Nobuhiro ; Ishida, Yasushi ; Imashuku, Shinsaku ; Kato, Shunichi ; Hara, Toshiro. / Hematopoietic stem cell transplantation for familial hemophagocytic lymphohistiocytosis and Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis in Japan. In: Pediatric Blood and Cancer. 2010 ; Vol. 54, No. 2. pp. 299-306.
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abstract = "Background. Post-transplant outcomes of hemophagocytic lymphohistiocytosis (HLH) patients were analyzed in Japan where Epstein-Barr virus (EBV)-associated severe forms are problematic. Methods. Fifty-seven patients (43 familial HLH [12 FHL2, 11 FHL3, 20 undefined], 14 EBV-HLH) who underwent stem cell transplantation (SCT) between 1995 and 2005 were enrolled based on the nationwide registration. Results. Fifty-seven patients underwent 61 SCTs, including 4 consecutive SCTs. SCTs were employed using allogeneic donors in 93{\%} of cases (allo 53, twin 1, auto 3). Unrelated donor cord blood transplantation (UCBT) was employed in half of cases (21 FHL, 7 EBV-HLH). Reduced intensity conditioning was used in 26{\%} of cases. The 10-year overall survival rates (median±SE{\%}) were 65.0±7.9{\%} in FHL and 85.7±9.4{\%} in EBV-HLH patients, respectively. The survival of UCBT recipients was >65{\%} in both FHL and EBV-HLH patients. Three out of four patients were alive with successful engraftment after second UCBT. FHL patients showed a poorer outcome due to early treatmentrelated deaths (<100 days, seven patients) and a higher incidence of sequelae than EBV-HLH patients (P=0.02). The risk of death for FHL patients having received an unrelated donor bone marrow transplant was marginally higher than that for a related donor SCT (P=0.05) and that for UCBT (P=0.07). Conclusions. EBV-HLH patients had a better prognosis after SCT than FHL patients. FHL patients showed either an equal or better outcome even after UCBT compared with the recent reports. UCB might therefore be acceptable as an alternate SCT source for HLH patients, although the optimal conditioning remains to be determined.",
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T1 - Hematopoietic stem cell transplantation for familial hemophagocytic lymphohistiocytosis and Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis in Japan

AU - Ohga, Shouichi

AU - Kudo, Kazuko

AU - Ishii, Eiichi

AU - Honjo, Satoshi

AU - Morimoto, Akira

AU - Osugi, Yuko

AU - Sawada, Akihisa

AU - Inoue, Masami

AU - Tabuchi, Ken

AU - Suzuki, Nobuhiro

AU - Ishida, Yasushi

AU - Imashuku, Shinsaku

AU - Kato, Shunichi

AU - Hara, Toshiro

PY - 2010/2/1

Y1 - 2010/2/1

N2 - Background. Post-transplant outcomes of hemophagocytic lymphohistiocytosis (HLH) patients were analyzed in Japan where Epstein-Barr virus (EBV)-associated severe forms are problematic. Methods. Fifty-seven patients (43 familial HLH [12 FHL2, 11 FHL3, 20 undefined], 14 EBV-HLH) who underwent stem cell transplantation (SCT) between 1995 and 2005 were enrolled based on the nationwide registration. Results. Fifty-seven patients underwent 61 SCTs, including 4 consecutive SCTs. SCTs were employed using allogeneic donors in 93% of cases (allo 53, twin 1, auto 3). Unrelated donor cord blood transplantation (UCBT) was employed in half of cases (21 FHL, 7 EBV-HLH). Reduced intensity conditioning was used in 26% of cases. The 10-year overall survival rates (median±SE%) were 65.0±7.9% in FHL and 85.7±9.4% in EBV-HLH patients, respectively. The survival of UCBT recipients was >65% in both FHL and EBV-HLH patients. Three out of four patients were alive with successful engraftment after second UCBT. FHL patients showed a poorer outcome due to early treatmentrelated deaths (<100 days, seven patients) and a higher incidence of sequelae than EBV-HLH patients (P=0.02). The risk of death for FHL patients having received an unrelated donor bone marrow transplant was marginally higher than that for a related donor SCT (P=0.05) and that for UCBT (P=0.07). Conclusions. EBV-HLH patients had a better prognosis after SCT than FHL patients. FHL patients showed either an equal or better outcome even after UCBT compared with the recent reports. UCB might therefore be acceptable as an alternate SCT source for HLH patients, although the optimal conditioning remains to be determined.

AB - Background. Post-transplant outcomes of hemophagocytic lymphohistiocytosis (HLH) patients were analyzed in Japan where Epstein-Barr virus (EBV)-associated severe forms are problematic. Methods. Fifty-seven patients (43 familial HLH [12 FHL2, 11 FHL3, 20 undefined], 14 EBV-HLH) who underwent stem cell transplantation (SCT) between 1995 and 2005 were enrolled based on the nationwide registration. Results. Fifty-seven patients underwent 61 SCTs, including 4 consecutive SCTs. SCTs were employed using allogeneic donors in 93% of cases (allo 53, twin 1, auto 3). Unrelated donor cord blood transplantation (UCBT) was employed in half of cases (21 FHL, 7 EBV-HLH). Reduced intensity conditioning was used in 26% of cases. The 10-year overall survival rates (median±SE%) were 65.0±7.9% in FHL and 85.7±9.4% in EBV-HLH patients, respectively. The survival of UCBT recipients was >65% in both FHL and EBV-HLH patients. Three out of four patients were alive with successful engraftment after second UCBT. FHL patients showed a poorer outcome due to early treatmentrelated deaths (<100 days, seven patients) and a higher incidence of sequelae than EBV-HLH patients (P=0.02). The risk of death for FHL patients having received an unrelated donor bone marrow transplant was marginally higher than that for a related donor SCT (P=0.05) and that for UCBT (P=0.07). Conclusions. EBV-HLH patients had a better prognosis after SCT than FHL patients. FHL patients showed either an equal or better outcome even after UCBT compared with the recent reports. UCB might therefore be acceptable as an alternate SCT source for HLH patients, although the optimal conditioning remains to be determined.

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