乳児期に非血縁者間臍帯血移植を施行したムコ多糖症I型 (Hurler症候群) の1例
症例は8カ月男児, ムコ多糖症1型 (Hurler症候群) に対して血清学的にHLA1座不一致の非血縁者間臍帯血移植 (UCBT) を施行した.前処置としてbusulfan35mg/m2×4/日×4日間 (day-9～-6), cyclophosphamide50mg/kg×4日間 (day-5～-2), 抗胸腺細胞グロブリン (ATG) 25mg/kg×4日間 (day-5～-2) を投与した.輸注細胞数は4.2×107/kgであった.白血球数>1,000/μ1, 血小板数>5万/μ1はそれぞれdayl6, day56であった.移植後3カ月時点での末梢血はドナータイプ78.6%,...
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| Published in | 日本小児血液学会雑誌 Vol. 14; no. 1; pp. 23 - 28 |
|---|---|
| Main Authors | , , , , , , , , |
| Format | Journal Article |
| Language | Japanese |
| Published |
特定非営利活動法人 日本小児血液・がん学会
29.02.2000
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| Subjects | |
| Online Access | Get full text |
| ISSN | 0913-8706 1884-4723 |
| DOI | 10.11412/jjph1987.14.23 |
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| Abstract | 症例は8カ月男児, ムコ多糖症1型 (Hurler症候群) に対して血清学的にHLA1座不一致の非血縁者間臍帯血移植 (UCBT) を施行した.前処置としてbusulfan35mg/m2×4/日×4日間 (day-9～-6), cyclophosphamide50mg/kg×4日間 (day-5～-2), 抗胸腺細胞グロブリン (ATG) 25mg/kg×4日間 (day-5～-2) を投与した.輸注細胞数は4.2×107/kgであった.白血球数>1,000/μ1, 血小板数>5万/μ1はそれぞれdayl6, day56であった.移植後3カ月時点での末梢血はドナータイプ78.6%, レシピエントタイプ21.4%のキメラ状態を呈したが, 移植後12カ月においてもその割合は安定していた.急性および慢性GVHDは認めなかった.末梢血リンパ球中α-iduronidaseおよび尿中ムコ多糖は移植後ほぼ正常範囲となった.早期の造血幹細胞移植を必要とするHurler症候群患児において, 家族内ドナーがいない場合UCBTは考慮されるべき治療法のひとつと考えられた. |
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| AbstractList | 症例は8カ月男児, ムコ多糖症1型 (Hurler症候群) に対して血清学的にHLA1座不一致の非血縁者間臍帯血移植 (UCBT) を施行した.前処置としてbusulfan35mg/m2×4/日×4日間 (day-9～-6), cyclophosphamide50mg/kg×4日間 (day-5～-2), 抗胸腺細胞グロブリン (ATG) 25mg/kg×4日間 (day-5～-2) を投与した.輸注細胞数は4.2×107/kgであった.白血球数>1,000/μ1, 血小板数>5万/μ1はそれぞれdayl6, day56であった.移植後3カ月時点での末梢血はドナータイプ78.6%, レシピエントタイプ21.4%のキメラ状態を呈したが, 移植後12カ月においてもその割合は安定していた.急性および慢性GVHDは認めなかった.末梢血リンパ球中α-iduronidaseおよび尿中ムコ多糖は移植後ほぼ正常範囲となった.早期の造血幹細胞移植を必要とするHurler症候群患児において, 家族内ドナーがいない場合UCBTは考慮されるべき治療法のひとつと考えられた. |
| Author | 古野, 憲司 稲光, 毅 西平, 浩一 住江, 愛子 井上, 和彦 井原, 健二 高田, 英俊 原, 寿郎 松崎, 彰信 |
| Author_FL | HARA Toshiro FURUNO Kenji NISHIHIRA Hirokazu INOUE Kazuhiko MATSUZAKI Akinobu INAMITSU Takeshi IHARA Kenji TAKADA Hidetoshi SUMINOE Aiko |
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| DocumentTitleAlternate | An Infant with Hurler Syndrome : Successful Engraftment after Unrelated Cord Blood Transplantation |
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| References | 13) 服部欽哉, 矢部普正, 矢部みはる, 他 : 臍帯血幹細胞移植を施行した急性骨髄性白血病 (M1.臨床血液37 : 1371-1376, L996 8) Gluckman E, Rocha V, Boyer-Chammard A, et al : Outcome of cord-blood transplantation from related and unrelated donors. Eurocord Transplant Group and the European Blood and Marrow Transplantation Group. N Engl J Med 337 : 373-381, 1997 14) Field RE, Buchanan JA, Copplemans MG, et al : Bone-marrow transplantation in Hurler's syndrome. Effect on skeletal development. J Bone Jt Surg Br Vol 76 : 975-981, 1994 11) Neufeld EF, Muenzer J : The Mucopolysaccharidoses. The Metabolic and Molecular Bases of Inherited Disease, Scriver CR ed 7th ed McGraw-Hill Inc. New York 1995, 2465-2494 2) Vellodi A, Young EP, Cooper A, et al : Bone marrow transplantation for mucopolysaccharidosis type I : Experience of two British centres. Arch Dis Child 76 : 92-99, 1997 19) 近藤達郎, 松本正, 天本なぎさ, 他 : 骨髄移植を施行したDiGeorge症候群における遺伝的, 免疫学的検討.血液系疾患調査研究班原発性免疫不全症候群分科会平成9年度研究業績報告書, 11-16, 1998 3) Guffon N, Souillet G, Maire I, et al : Follow-up of nine patients with Hurler syndrome after bone marrow transplantation. J Pediatr 133 : 119-125, 1998 4) Peters C, Shapiro EG, Anderson J, et al : Hurler syndrome : II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group. Blood 91 : 2601-2608, 1998 6) Kurtzberg J, Laughlin M, Graham ML, et al : Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients. N Engl J Med 335 : 157-166, 1996 5) Wagner JE, Kernan NA, Steinbuch M, et al : Allogeneic sibling umbilical-cord-blood transplantation in children with malignant and non-malignant disease. Lancet 346 : 214-219, 1995 7) Wagner JE, Rosenthal J, Sweetman R, et al : Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donors : Analysis of engraftment and acute graftversus-host disease. Blood 88 : 795-802, 1996 16) Odunusi E, Peters C, Krivit W, et al : Genu valgum deformity in Hurler syndrome after hematopoietic stem cell transplantation : Correction by surgical intervention. J Pediatr Orthop 19 : 270-274, 1999 17) Foot AB, Potter MN, Donaldson C, et al : Immune reconstitution after BMT in children. Bone Marrow Transplant 11 : 7-13, 1993 10) Rubinstein P, Carrier C, Scaradavou A, et al : Outcomes among 562 recipients of placental-blood transplants from unrelated donors. N Engl J Med 339 : 1565-1577, 1998 15) Van Heest AE, House J, Krivit W, et al : Surgical treatment of carpal tunnel syndrome and trigger digits in children with mucopolysaccharide storage disorders. J Hand Surg 23 : 236-243, 1998 12) Gluckman E, Broxmeyer HE, Auerbach AD, et al : Hematopoietic reconstitution in a patient with Fanconi anemia by means of umbilical-cord blood from an HLA-identical sibling. N Engl J Med 321 : 1174-1178, 1989 1) Peters C, Balthazor M, Shapiro EG, et al : Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood 87 : 4894-4902, 1996 9) Cairo MS, Wagner JE : Placental and/or umbilical cord blood : An alternative source of hematopoietic stem cells for transplantation. Blood 90 : 4665-4678, 1997 18) Locatelli F, Maccario R, Comoli P, et al : Hematopoietic and immune recovery after transplantation of cord blood progenitor cells in children. Bone Marrow Transplant 18 : 1095-1101, 1996 |
| References_xml | – reference: 3) Guffon N, Souillet G, Maire I, et al : Follow-up of nine patients with Hurler syndrome after bone marrow transplantation. J Pediatr 133 : 119-125, 1998 – reference: 17) Foot AB, Potter MN, Donaldson C, et al : Immune reconstitution after BMT in children. Bone Marrow Transplant 11 : 7-13, 1993 – reference: 2) Vellodi A, Young EP, Cooper A, et al : Bone marrow transplantation for mucopolysaccharidosis type I : Experience of two British centres. Arch Dis Child 76 : 92-99, 1997 – reference: 18) Locatelli F, Maccario R, Comoli P, et al : Hematopoietic and immune recovery after transplantation of cord blood progenitor cells in children. Bone Marrow Transplant 18 : 1095-1101, 1996 – reference: 12) Gluckman E, Broxmeyer HE, Auerbach AD, et al : Hematopoietic reconstitution in a patient with Fanconi anemia by means of umbilical-cord blood from an HLA-identical sibling. N Engl J Med 321 : 1174-1178, 1989 – reference: 7) Wagner JE, Rosenthal J, Sweetman R, et al : Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donors : Analysis of engraftment and acute graftversus-host disease. Blood 88 : 795-802, 1996 – reference: 9) Cairo MS, Wagner JE : Placental and/or umbilical cord blood : An alternative source of hematopoietic stem cells for transplantation. Blood 90 : 4665-4678, 1997 – reference: 13) 服部欽哉, 矢部普正, 矢部みはる, 他 : 臍帯血幹細胞移植を施行した急性骨髄性白血病 (M1.臨床血液37 : 1371-1376, L996 – reference: 15) Van Heest AE, House J, Krivit W, et al : Surgical treatment of carpal tunnel syndrome and trigger digits in children with mucopolysaccharide storage disorders. J Hand Surg 23 : 236-243, 1998 – reference: 16) Odunusi E, Peters C, Krivit W, et al : Genu valgum deformity in Hurler syndrome after hematopoietic stem cell transplantation : Correction by surgical intervention. J Pediatr Orthop 19 : 270-274, 1999 – reference: 6) Kurtzberg J, Laughlin M, Graham ML, et al : Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients. N Engl J Med 335 : 157-166, 1996 – reference: 1) Peters C, Balthazor M, Shapiro EG, et al : Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood 87 : 4894-4902, 1996 – reference: 14) Field RE, Buchanan JA, Copplemans MG, et al : Bone-marrow transplantation in Hurler's syndrome. Effect on skeletal development. J Bone Jt Surg Br Vol 76 : 975-981, 1994 – reference: 10) Rubinstein P, Carrier C, Scaradavou A, et al : Outcomes among 562 recipients of placental-blood transplants from unrelated donors. N Engl J Med 339 : 1565-1577, 1998 – reference: 11) Neufeld EF, Muenzer J : The Mucopolysaccharidoses. The Metabolic and Molecular Bases of Inherited Disease, Scriver CR ed 7th ed McGraw-Hill Inc. New York 1995, 2465-2494 – reference: 8) Gluckman E, Rocha V, Boyer-Chammard A, et al : Outcome of cord-blood transplantation from related and unrelated donors. Eurocord Transplant Group and the European Blood and Marrow Transplantation Group. N Engl J Med 337 : 373-381, 1997 – reference: 4) Peters C, Shapiro EG, Anderson J, et al : Hurler syndrome : II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group. Blood 91 : 2601-2608, 1998 – reference: 5) Wagner JE, Kernan NA, Steinbuch M, et al : Allogeneic sibling umbilical-cord-blood transplantation in children with malignant and non-malignant disease. Lancet 346 : 214-219, 1995 – reference: 19) 近藤達郎, 松本正, 天本なぎさ, 他 : 骨髄移植を施行したDiGeorge症候群における遺伝的, 免疫学的検討.血液系疾患調査研究班原発性免疫不全症候群分科会平成9年度研究業績報告書, 11-16, 1998 |
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| Snippet | 症例は8カ月男児, ムコ多糖症1型 (Hurler症候群) に対して血清学的にHLA1座不一致の非血縁者間臍帯血移植 (UCBT) を施行した.前処置としてbusulfan35mg/m2×4/日×4日間 (day-9～-6), cyclophosphamide50mg/kg×4日間 (day-5～-2),... |
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| Title | 乳児期に非血縁者間臍帯血移植を施行したムコ多糖症I型 (Hurler症候群) の1例 |
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