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 |
| 巻 | 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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All Science Journal Classification (ASJC) codes
- Hematology
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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
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TY - JOUR
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
ER -