Reduced-intensity conditioning in unrelated donor cord blood transplantation for familial hemophagocytic lymphohistiocytosis

Masanori Nishi, Ryosei Nishimura, Nobuhiro Suzuki, Akihisa Sawada, Takayuki Okamura, Naoto Fujita, Rie Kanai, Jun Yano, Souichi Adachi, Takahiro Yasumi, Emiko Sato, Koji Yasutomo, Eiichi Ishii, Shoichi Ohga

研究成果: ジャーナルへの寄稿レター

24 引用 (Scopus)

抄録

Familial hemophagocytic lymphohistiocytosis (FHL) is a disorder of immune homeostasis characterized by fever, cytopenias, hepatosplenomegaly, and coagulopathy. We studied the outcomes of 13 FHL patients who underwent the first unrelated cord blood transplantation (UCBT) after non-myeloablative conditionings. The major regimen consisted of fludarabine (FLU; n = 12)+melphalan (MEL; n = 11)±low-dose total body irradiation (TBI 2-4 Gy; n = 6). The median age at presentation and period to UCBT were 6 and 5 months, respectively. Central nervous system (CNS) disease developed in one infant at diagnosis, and in two others until UCBT. HLH activity was controlled in all but one at the time of UCBT. Ten patients had early engraftment on median day 21 with no grade >2 treatment-related toxicity and two controllable grade >2 acute GVHD. Two patients with early rejection successfully underwent subsequent UCBT after myeloablative conditioning. Two others had late graft failure following mixed donor chimerism. Two deaths occurred from HLH; early liver failure and late CNS disease. Of 11 FLU+MEL-conditioned patients, the frequency of disease-free complete engraftment was higher for MEL (≥120 mg/m 2 )+TBI, or high-dose MEL (180 mg/m 2 ) than for others (83% vs. 25%, p = 0.036). The FLU+MEL-based non-myeloablative regimen was acceptable for FHL infants undergoing UCBT, although further studies will be needed for confirmation.

元の言語英語
ページ(範囲)637-639
ページ数3
ジャーナルAmerican Journal of Hematology
87
発行部数6
DOI
出版物ステータス出版済み - 6 1 2012

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Hemophagocytic Lymphohistiocytosis
Unrelated Donors
Fetal Blood
Transplantation
Central Nervous System Diseases
Chimerism
Melphalan
Whole-Body Irradiation
Immune System Diseases
Liver Failure
Homeostasis
Fever
Tissue Donors
Transplants

All Science Journal Classification (ASJC) codes

  • Hematology

これを引用

Reduced-intensity conditioning in unrelated donor cord blood transplantation for familial hemophagocytic lymphohistiocytosis. / Nishi, Masanori; Nishimura, Ryosei; Suzuki, Nobuhiro; Sawada, Akihisa; Okamura, Takayuki; Fujita, Naoto; Kanai, Rie; Yano, Jun; Adachi, Souichi; Yasumi, Takahiro; Sato, Emiko; Yasutomo, Koji; Ishii, Eiichi; Ohga, Shoichi.

:: American Journal of Hematology, 巻 87, 番号 6, 01.06.2012, p. 637-639.

研究成果: ジャーナルへの寄稿レター

Nishi, M, Nishimura, R, Suzuki, N, Sawada, A, Okamura, T, Fujita, N, Kanai, R, Yano, J, Adachi, S, Yasumi, T, Sato, E, Yasutomo, K, Ishii, E & Ohga, S 2012, 'Reduced-intensity conditioning in unrelated donor cord blood transplantation for familial hemophagocytic lymphohistiocytosis', American Journal of Hematology, 巻. 87, 番号 6, pp. 637-639. https://doi.org/10.1002/ajh.23190
Nishi, Masanori ; Nishimura, Ryosei ; Suzuki, Nobuhiro ; Sawada, Akihisa ; Okamura, Takayuki ; Fujita, Naoto ; Kanai, Rie ; Yano, Jun ; Adachi, Souichi ; Yasumi, Takahiro ; Sato, Emiko ; Yasutomo, Koji ; Ishii, Eiichi ; Ohga, Shoichi. / Reduced-intensity conditioning in unrelated donor cord blood transplantation for familial hemophagocytic lymphohistiocytosis. :: American Journal of Hematology. 2012 ; 巻 87, 番号 6. pp. 637-639.
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abstract = "Familial hemophagocytic lymphohistiocytosis (FHL) is a disorder of immune homeostasis characterized by fever, cytopenias, hepatosplenomegaly, and coagulopathy. We studied the outcomes of 13 FHL patients who underwent the first unrelated cord blood transplantation (UCBT) after non-myeloablative conditionings. The major regimen consisted of fludarabine (FLU; n = 12)+melphalan (MEL; n = 11)±low-dose total body irradiation (TBI 2-4 Gy; n = 6). The median age at presentation and period to UCBT were 6 and 5 months, respectively. Central nervous system (CNS) disease developed in one infant at diagnosis, and in two others until UCBT. HLH activity was controlled in all but one at the time of UCBT. Ten patients had early engraftment on median day 21 with no grade >2 treatment-related toxicity and two controllable grade >2 acute GVHD. Two patients with early rejection successfully underwent subsequent UCBT after myeloablative conditioning. Two others had late graft failure following mixed donor chimerism. Two deaths occurred from HLH; early liver failure and late CNS disease. Of 11 FLU+MEL-conditioned patients, the frequency of disease-free complete engraftment was higher for MEL (≥120 mg/m 2 )+TBI, or high-dose MEL (180 mg/m 2 ) than for others (83{\%} vs. 25{\%}, p = 0.036). The FLU+MEL-based non-myeloablative regimen was acceptable for FHL infants undergoing UCBT, although further studies will be needed for confirmation.",
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T1 - Reduced-intensity conditioning in unrelated donor cord blood transplantation for familial hemophagocytic lymphohistiocytosis

AU - Nishi, Masanori

AU - Nishimura, Ryosei

AU - Suzuki, Nobuhiro

AU - Sawada, Akihisa

AU - Okamura, Takayuki

AU - Fujita, Naoto

AU - Kanai, Rie

AU - Yano, Jun

AU - Adachi, Souichi

AU - Yasumi, Takahiro

AU - Sato, Emiko

AU - Yasutomo, Koji

AU - Ishii, Eiichi

AU - Ohga, Shoichi

PY - 2012/6/1

Y1 - 2012/6/1

N2 - Familial hemophagocytic lymphohistiocytosis (FHL) is a disorder of immune homeostasis characterized by fever, cytopenias, hepatosplenomegaly, and coagulopathy. We studied the outcomes of 13 FHL patients who underwent the first unrelated cord blood transplantation (UCBT) after non-myeloablative conditionings. The major regimen consisted of fludarabine (FLU; n = 12)+melphalan (MEL; n = 11)±low-dose total body irradiation (TBI 2-4 Gy; n = 6). The median age at presentation and period to UCBT were 6 and 5 months, respectively. Central nervous system (CNS) disease developed in one infant at diagnosis, and in two others until UCBT. HLH activity was controlled in all but one at the time of UCBT. Ten patients had early engraftment on median day 21 with no grade >2 treatment-related toxicity and two controllable grade >2 acute GVHD. Two patients with early rejection successfully underwent subsequent UCBT after myeloablative conditioning. Two others had late graft failure following mixed donor chimerism. Two deaths occurred from HLH; early liver failure and late CNS disease. Of 11 FLU+MEL-conditioned patients, the frequency of disease-free complete engraftment was higher for MEL (≥120 mg/m 2 )+TBI, or high-dose MEL (180 mg/m 2 ) than for others (83% vs. 25%, p = 0.036). The FLU+MEL-based non-myeloablative regimen was acceptable for FHL infants undergoing UCBT, although further studies will be needed for confirmation.

AB - Familial hemophagocytic lymphohistiocytosis (FHL) is a disorder of immune homeostasis characterized by fever, cytopenias, hepatosplenomegaly, and coagulopathy. We studied the outcomes of 13 FHL patients who underwent the first unrelated cord blood transplantation (UCBT) after non-myeloablative conditionings. The major regimen consisted of fludarabine (FLU; n = 12)+melphalan (MEL; n = 11)±low-dose total body irradiation (TBI 2-4 Gy; n = 6). The median age at presentation and period to UCBT were 6 and 5 months, respectively. Central nervous system (CNS) disease developed in one infant at diagnosis, and in two others until UCBT. HLH activity was controlled in all but one at the time of UCBT. Ten patients had early engraftment on median day 21 with no grade >2 treatment-related toxicity and two controllable grade >2 acute GVHD. Two patients with early rejection successfully underwent subsequent UCBT after myeloablative conditioning. Two others had late graft failure following mixed donor chimerism. Two deaths occurred from HLH; early liver failure and late CNS disease. Of 11 FLU+MEL-conditioned patients, the frequency of disease-free complete engraftment was higher for MEL (≥120 mg/m 2 )+TBI, or high-dose MEL (180 mg/m 2 ) than for others (83% vs. 25%, p = 0.036). The FLU+MEL-based non-myeloablative regimen was acceptable for FHL infants undergoing UCBT, although further studies will be needed for confirmation.

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U2 - 10.1002/ajh.23190

DO - 10.1002/ajh.23190

M3 - Letter

VL - 87

SP - 637

EP - 639

JO - American Journal of Hematology

JF - American Journal of Hematology

SN - 0361-8609

IS - 6

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