Nonmyeloablative Bone Marrow Transplant from HLA-Identical Sibling for Chronic Granulomatous Disease
Chronic granulomatous disease (CGD) is an inherited disorder caused by the abnormalities of NADPH oxidase in phagocytic cells. Stem cell transplantation (SCT) from an HLA-identical donor is an effective treatment for patients with CGD. However, SCT with myeloablative conditioning carries a high inci...
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| Published in | The Japanese Journal of Pediatric Hematology Vol. 20; no. 1; pp. 34 - 38 |
|---|---|
| Main Authors | , , , , , |
| Format | Journal Article |
| Language | Japanese |
| Published |
THE JAPANESE SOCIETY OF PEDIATRIC HEMATOLOGY/ONCOLOGY
2006
特定非営利活動法人 日本小児血液・がん学会 |
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| ISSN | 0913-8706 1884-4723 |
| DOI | 10.11412/jjph1987.20.34 |
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| Abstract | Chronic granulomatous disease (CGD) is an inherited disorder caused by the abnormalities of NADPH oxidase in phagocytic cells. Stem cell transplantation (SCT) from an HLA-identical donor is an effective treatment for patients with CGD. However, SCT with myeloablative conditioning carries a high incidence of serious complications and death. We report a 21-year-old man with CGD who was successfully treated with allogeneic bone marrow transplantation (alloBMT) from his HLA-identical brother following a nonmyeloablative conditioning regimen consisting of cyclophosphamide, fludarabine and a low dose of total body irradiation. Engraftment was rapidly achieved without any toxicity or complication. Partial donor chimerism following alloBMT was converted to complete donor chimerism and nomal oxidase activity of neutrophils was induced by several donor lymphocyte infusions (DLI) from his HLA-identical brother. At 30 months after transplant, the patient is in excellent clinical and hematological condition, maintaining complete chimerism. We suggest that alloBMT preceded by nonmyeloablative conditioning and followed by DLI may constitute a successful mode of therapy for patients with CGD. |
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| AbstractList | Chronic granulomatous disease (CGD) is an inherited disorder caused by the abnormalities of NADPH oxidase in phagocytic cells. Stem cell transplantation (SCT) from an HLA-identical donor is an effective treatment for patients with CGD. However, SCT with myeloablative conditioning carries a high incidence of serious complications and death. We report a 21-year-old man with CGD who was successfully treated with allogeneic bone marrow transplantation (alloBMT) from his HLA-identical brother following a nonmyeloablative conditioning regimen consisting of cyclophosphamide, fludarabine and a low dose of total body irradiation. Engraftment was rapidly achieved without any toxicity or complication. Partial donor chimerism following alloBMT was converted to complete donor chimerism and nomal oxidase activity of neutrophils was induced by several donor lymphocyte infusions (DLI) from his HLA-identical brother. At 30 months after transplant, the patient is in excellent clinical and hematological condition, maintaining complete chimerism. We suggest that alloBMT preceded by nonmyeloablative conditioning and followed by DLI may constitute a successful mode of therapy for patients with CGD. Chronic granulomatous disease (CGD) is an inherited disorder caused by the abnormalities of NADPH oxidase in phagocytic cells. Stem cell transplantation (SCT) from an HLA-identical donor is an effective treatment for patients with CGD. However, SCT with myeloablative conditioning carries a high incidence of serious complications and death. We report a 21-year-old man with CGD who was successfully treated with allogeneic bone marrow transplantation (alloBMT) from his HLA-identical brother following a nonmyeloablative conditioning regimen consisting of cyclophosphamide, fludarabine and a low dose of total body irradiation. Engraftment was rapidly achieved without any toxicity or complication. Partial donor chimerism following alloBMT was converted to complete donor chimerism and nomal oxidase activity of neutrophils was induced by several donor lymphocyte infusions (DLI) from his HLA-identical brother. At 30 months after transplant, the patient is in excellent clinical and hematological condition, maintaining complete chimerism. We suggest that alloBMT preceded by nonmyeloablative conditioning and followed by DLI may constitute a successful mode of therapy for patients with CGD. 慢性肉芽腫症 (CGD) は好中球の活性酸素産生障害により易感染性を呈する遺伝性疾患である.その根治療法として造血幹細胞移植があるが, 本疾患では潜在的感染や臓器予備能の低下により, 従来の骨髄破壊的前処置では治療関連毒性, 死亡のリスクが大きいとされる.今回われわれはCGDの21歳男性に対し, fludarabine, cyclophosphamide, 全身放射線照射 (TBI) による骨髄非破壊的前処置を選択し, HLA完全一致の兄より同種骨髄移植を施行した.治療関連毒性はなく, 生着はすみやかであった.混合キメラの状態であったが, 4回のドナーリンパ球輸注 (DLI) を施行し, 移植後1年で完全キメラを達成した.移植後30カ月を経過し, 好中球の活性酸素産生能は正常に維持され良好な経過を得ている.慢性肉芽腫症の根治療法として骨髄非破壊的骨髄移植は有効な治療法であると考えられた. |
| Author | ONO, Mayuko FUJITA, Naoto MIZUKAMI, Tomoyuki HAMAMOTO, Kazuko KOBAYASHI, Masao NUNOI, Hiroyuki |
| Author_FL | 小野 麻由子 小林 正夫 布井 博幸 藤田 直人 水上 智之 浜本 和子 |
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| References | 3) 水上智之, 石橋史成, 布井博幸 : 慢性肉芽腫症の病因・病態と治療.小児内科32 : 2032-2035, 2000 2) 崎山幸雄 : 原発性免疫不全症の根治療法.小児内科32 : 1975-1979, 2000 8) Goudemand J, Anssens R, Delmas-Marsalet Y, et al : Attempt to treat a case of chronic familial granulomatous disease by allogeneic bone marrow transplantation. Arch Fr Pediatr 33 : 121-129, 1976 9) Leung T, Chik K, Shing M, et al : Bone marrow transplantation for chronic granulomatous disease : Long term follow up and review of literature. Bone Marrow Transplant 24 : 567-570, 1999 22) 大西敏弘, 河野嘉文 : *同種末梢血幹細胞移植術を施行した慢性肉芽腫症の1例.小児感染免疫11 : 199, 1999 5) Yamauchi A, Yu L, Potgens AJ, et al : Location of the epitope for 7D5, a monoclonal antibody raised against human flavocytochrome b558, to the extracellular peptide portion of primate gp91 Phox. Microbiol Immunol 45 : 249-257, 2001 14) Horwitz ME, Barrett AJ, Brown MR, et al : Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft. N Engl J Med 344 : 881-888, 2001 10) Del Giudice I, Iori AP, Mengarelli A, et al : Allogeneic stem cell transplant from HLA-identical sibling for chronic granulomatous disease and review of the literature. Ann Hematol 82 : 189-192, 2003 11) Ozsahin H, von Planta M, Muller I, et al : Successful treatment of invasive aspergillosis in chronic granulomatous disease by bone marrow transplantation, granulocyte colony-stimulating factor-mobilized granuocytes, and liposomal amphotericin-B. Blood 92 : 2719-2724, 1998 17) 新谷いつみ, 田内久道, 石田也寸志, 他 : *多発性肝膿瘍を合併した慢性肉芽腫症に対するミニ移植の試み.愛媛医学22 : 270, 2003 13) Nagler A, Ackerstein A, Kapelushnik J, et al : Donor lymphocyte infusion post-non-myeloablative allogeneic peripheral blood stem cell transplantation for chronic granulomatous disease. Bone Marrow Transplant 24 : 339-342, 1999 7) 康勝好, 上山潤一, 菊地陽, 他 : HLAI座不一致の同胞より同種骨髄移植を施行した慢性肉芽腫症 (CGD) の一例.埼玉小児医療センター医学誌18 : 31-35, 2001 23) 上山潤一, 康勝好, 菊地陽, 他 : *同種骨髄移植を施行した慢性肉芽腫症の2例.日小血会誌16 : 266, 2002 4) 小林信一, 立澤宰 : 食細胞機能異常症.小児内科36 : 1792-1798, 2004 6) Vowells SJ, Sekhsaria S, Malech HL, et al : Flow cytometric analysis of the granulocyte respiratory burst : A comparison study of fluorescent probes. J Immunol Methods 178 : 89-97, 1995 21) 鹿間芳明, 奥山信彦, 赤城邦彦, 他 : *骨髄移植した慢性肉芽腫症の経過.小児感染免疫11 : 199, 1999 1) 布井博幸, 石橋史成 : 日本の慢性肉芽腫症の現状と将来.臨床病理47 : 658-664, 1999 20) 出口靖, 小林信一, 永田正人, 他 : *慢性肉芽腫症の骨髄移植で見られたGVHD様経過.小児感染免疫11 : 199, 1999 16) 食細胞機能異常症研究会BMTアンケート調査グループ : BMTアンケート調査.小児感染免疫12 : 254, 2000 19) 朴永東, 中野智巳, 吉岡章, 他 : *非血縁者間膀帯血移植を施行したMcLeod表現型の慢性肉芽腫症の一例.小児感染免疫11 : 198-199, 1999 18) 藤原亨, 山田実名美, 遠宮靖雄, 他 : *骨髄破壊的同種末梢血幹細胞移植 (NST) を施行した慢性肉芽腫症 (CGD) の1例.臨床血液44 : 746, 2003 15) 日本小児血液学会造血幹細胞移植委員会 : 小児期造血幹細胞移植全国集計 (1998).日小血会誌14 : 333-337, 2000 12) Seger RA, Gungor T, Belohradsky BH, et al : Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft : A survey of the European experience, 1985-2000. Blood 100 : 4344-4350, 2002 |
| References_xml | – reference: 2) 崎山幸雄 : 原発性免疫不全症の根治療法.小児内科32 : 1975-1979, 2000 – reference: 6) Vowells SJ, Sekhsaria S, Malech HL, et al : Flow cytometric analysis of the granulocyte respiratory burst : A comparison study of fluorescent probes. J Immunol Methods 178 : 89-97, 1995 – reference: 7) 康勝好, 上山潤一, 菊地陽, 他 : HLAI座不一致の同胞より同種骨髄移植を施行した慢性肉芽腫症 (CGD) の一例.埼玉小児医療センター医学誌18 : 31-35, 2001 – reference: 21) 鹿間芳明, 奥山信彦, 赤城邦彦, 他 : *骨髄移植した慢性肉芽腫症の経過.小児感染免疫11 : 199, 1999 – reference: 12) Seger RA, Gungor T, Belohradsky BH, et al : Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft : A survey of the European experience, 1985-2000. Blood 100 : 4344-4350, 2002 – reference: 4) 小林信一, 立澤宰 : 食細胞機能異常症.小児内科36 : 1792-1798, 2004 – reference: 18) 藤原亨, 山田実名美, 遠宮靖雄, 他 : *骨髄破壊的同種末梢血幹細胞移植 (NST) を施行した慢性肉芽腫症 (CGD) の1例.臨床血液44 : 746, 2003 – reference: 5) Yamauchi A, Yu L, Potgens AJ, et al : Location of the epitope for 7D5, a monoclonal antibody raised against human flavocytochrome b558, to the extracellular peptide portion of primate gp91 Phox. Microbiol Immunol 45 : 249-257, 2001 – reference: 17) 新谷いつみ, 田内久道, 石田也寸志, 他 : *多発性肝膿瘍を合併した慢性肉芽腫症に対するミニ移植の試み.愛媛医学22 : 270, 2003 – reference: 10) Del Giudice I, Iori AP, Mengarelli A, et al : Allogeneic stem cell transplant from HLA-identical sibling for chronic granulomatous disease and review of the literature. Ann Hematol 82 : 189-192, 2003 – reference: 22) 大西敏弘, 河野嘉文 : *同種末梢血幹細胞移植術を施行した慢性肉芽腫症の1例.小児感染免疫11 : 199, 1999 – reference: 14) Horwitz ME, Barrett AJ, Brown MR, et al : Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft. N Engl J Med 344 : 881-888, 2001 – reference: 3) 水上智之, 石橋史成, 布井博幸 : 慢性肉芽腫症の病因・病態と治療.小児内科32 : 2032-2035, 2000 – reference: 20) 出口靖, 小林信一, 永田正人, 他 : *慢性肉芽腫症の骨髄移植で見られたGVHD様経過.小児感染免疫11 : 199, 1999 – reference: 8) Goudemand J, Anssens R, Delmas-Marsalet Y, et al : Attempt to treat a case of chronic familial granulomatous disease by allogeneic bone marrow transplantation. Arch Fr Pediatr 33 : 121-129, 1976 – reference: 19) 朴永東, 中野智巳, 吉岡章, 他 : *非血縁者間膀帯血移植を施行したMcLeod表現型の慢性肉芽腫症の一例.小児感染免疫11 : 198-199, 1999 – reference: 9) Leung T, Chik K, Shing M, et al : Bone marrow transplantation for chronic granulomatous disease : Long term follow up and review of literature. Bone Marrow Transplant 24 : 567-570, 1999 – reference: 16) 食細胞機能異常症研究会BMTアンケート調査グループ : BMTアンケート調査.小児感染免疫12 : 254, 2000 – reference: 23) 上山潤一, 康勝好, 菊地陽, 他 : *同種骨髄移植を施行した慢性肉芽腫症の2例.日小血会誌16 : 266, 2002 – reference: 1) 布井博幸, 石橋史成 : 日本の慢性肉芽腫症の現状と将来.臨床病理47 : 658-664, 1999 – reference: 13) Nagler A, Ackerstein A, Kapelushnik J, et al : Donor lymphocyte infusion post-non-myeloablative allogeneic peripheral blood stem cell transplantation for chronic granulomatous disease. Bone Marrow Transplant 24 : 339-342, 1999 – reference: 11) Ozsahin H, von Planta M, Muller I, et al : Successful treatment of invasive aspergillosis in chronic granulomatous disease by bone marrow transplantation, granulocyte colony-stimulating factor-mobilized granuocytes, and liposomal amphotericin-B. Blood 92 : 2719-2724, 1998 – reference: 15) 日本小児血液学会造血幹細胞移植委員会 : 小児期造血幹細胞移植全国集計 (1998).日小血会誌14 : 333-337, 2000 |
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| Snippet | Chronic granulomatous disease (CGD) is an inherited disorder caused by the abnormalities of NADPH oxidase in phagocytic cells. Stem cell transplantation (SCT)... |
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| SubjectTerms | bone marrow transplantation chronic granulomatous disease donor lymphocyte infusion nonmyeloablative stem cell transplantation |
| Title | Nonmyeloablative Bone Marrow Transplant from HLA-Identical Sibling for Chronic Granulomatous Disease |
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