Clinical efficacy of haematopoietic stem cell transplantation for adult adrenoleukodystrophy

被引：19

作者：

Matsukawa, Takashi ^{[1
,2
]}

Yamamoto, Tomotaka ^{[1
]}

Honda, Akira ^{[3
]}

Toya, Takashi ^{[3
]}

Ishiura, Hiroyuki ^{[1
]}

Mitsui, Jun ^{[1
,2
]}

Tanaka, Masaki ^{[1
]}

Hao, Akihito ^{[1
]}

Shinohara, Akihito ^{[3
]}

Ogura, Mizuki ^{[3
]}

Kataoka, Keisuke ^{[3
]}

Seo, Sachiko ^{[3
]}

Kumano, Keiki ^{[3
]}

Hosoi, Masataka ^{[3
]}

Narukawa, Kensuke ^{[3
]}

Yasunaga, Megumi ^{[3
]}

Maki, Hiroaki ^{[3
]}

Ichikawa, Motoshi ^{[3
]}

Nannya, Yasuhito ^{[3
]}

Imai, Yoichi ^{[3
]}

Takahashi, Tsuyoshi ^{[3
]}

Takahashi, Yuji ^{[1
]}

Nagasako, Yuki ^{[1
]}

Yasaka, Kyoko ^{[1
]}

Mano, Kagari Koshi ^{[1
]}

Matsukawa, Miho Kawabe ^{[1
]}

Miyagawa, Toji ^{[1
]}

Hamada, Masashi ^{[1
]}

Sakuishi, Kaori ^{[1
]}

Hayashi, Toshihiro ^{[1
]}

Iwata, Atsushi ^{[1
]}

Terao, Yasuo ^{[1
]}

Shimizu, Jun ^{[1
]}

Goto, Jun ^{[1
,4
]}

Mori, Harushi ^{[5
]}

Kunimatsu, Akira ^{[5
]}

Aoki, Shigeki ^{[5
]}

Hayashi, Shin ^{[6
,7
]}

Nakamura, Fumihiko ^{[3
]}

Arai, Syunya ^{[3
]}

Momma, Kazunari ^{[8
]}

Ogata, Katsuhisa ^{[8
]}

Yoshida, Toshikazu ^{[9
]}

Abe, Osamu ^{[5
]}

Inazawa, Johji ^{[6
,7
]}

Toda, Tatsushi ^{[1
]}

Kurokawa, Mineo ^{[3
]}

Tsuji, Shoji ^{[1
,2
,10
]}

机构：

[1] Univ Tokyo, Grad Sch Med, Dept Neurol, Tokyo 1138655, Japan

[2] Univ Tokyo, Grad Sch Med, Dept Mol Neurol, Tokyo 1138655, Japan

[3] Univ Tokyo, Grad Sch Med, Dept Hematol & Oncol, Tokyo 1138655, Japan

[4] Int Univ Hlth & Welf, Mita Hosp, Dept Neurol, Tokyo 1088329, Japan

[5] Univ Tokyo, Grad Sch Med, Dept Radiol, Tokyo 1138655, Japan

[6] Tokyo Med & Dent Univ, Med Res Inst, Dept Mol Cytogenet, Tokyo 1138519, Japan

[7] Tokyo Med & Dent Univ, Sch Biomed Sci, Tokyo 1138519, Japan

[8] Natl Hosp Org Higashisaitama Natl Hosp, Dept Neurol, Saitama 3490196, Japan

[9] Fujimi Kogen Hosp, Dept Neurol, Nagano 3990214, Japan

[10] Int Univ Hlth & Welf, Chiba 2868686, Japan

来源：

BRAIN COMMUNICATIONS | 2020年 / 2卷 / 01期

关键词：

adrenoleukodystrophy; haematopoietic stem cell transplantation; nonmyeloablative preparative regimen; adult cerebral form; cerebello-brainstem form; X-LINKED ADRENOLEUKODYSTROPHY; BONE-MARROW-TRANSPLANTATION; OUTCOMES; ABNORMALITY;

D O I：

10.1093/braincomms/fcz048

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Accumulated experience supports the efficacy of allogenic haematopoietic stem cell transplantation in arresting the progression of childhood-onset cerebral form of adrenoleukodystrophy in early stages. For adulthood-onset cerebral form of adrenoleukodystrophy, however, there have been only a few reports on haematopoietic stem cell transplantation and the clinical efficacy and safety of that for adulthood-onset cerebral form of adrenoleukodystrophy remain to be established. To evaluate the clinical efficacy and safety of haematopoietic stem cell transplantation, we conducted haematopoietic stem cell transplantation on 12 patients with adolescent-/adult-onset cerebral form/cerebello-brainstem form of adrenoleukodystrophy in a single-institution-based prospective study. Through careful prospective follow-up of 45 male adrenoleukodystrophy patients, we aimed to enrol patients with adolescent-/adult-onset cerebral form/cerebello-brainstem form of adrenoleukodystrophy at early stages. Indications for haematopoietic stem cell transplantation included cerebral form of adrenoleukodystrophy or cerebello-brainstem form of adrenoleukodystrophy with Loes scores up to 13, the presence of progressively enlarging white matter lesions and/or lesions with gadolinium enhancement on brain MRI. Clinical outcomes of haematopoietic stem cell transplantation were evaluated by the survival rate as well as by serial evaluation of clinical rating scale scores and neurological and MRI findings. Clinical courses of eight patients who did not undergo haematopoietic stem cell transplantation were also evaluated for comparison of the survival rate. All the patients who underwent haematopoietic stem cell transplantation survived to date with a median follow-up period of 28.6 months (4.2-125.3 months) without fatality. Neurological findings attributable to cerebral/cerebellar/brainstem lesions became stable or partially improved in all the patients. Gadolinium-enhanced brain lesions disappeared or became obscure within 3.5 months and the white matter lesions of MRI became stable or small. The median Loes scores before haematopoietic stem cell transplantation and at the last follow-up visit were 6.0 and 5.25, respectively. Of the eight patients who did not undergo haematopoietic stem cell transplantation, six patients died 69.1 months (median period; range 16.0-104.1 months) after the onset of the cerebral/cerebellar/brainstem lesions, confirming that the survival probability was significantly higher in patients with haematopoietic stem cell transplantation compared with that in patients without haematopoietic stem cell transplantation (P = 0.0089). The present study showed that haematopoietic stem cell transplantation was conducted safely and arrested the inflammatory demyelination in all the patients with adolescent-/adult-onset cerebral form/cerebello-brainstem form of adrenoleukodystrophy when haematopoietic stem cell transplantation was conducted in the early stages. Further studies are warranted to optimize the procedures of haematopoietic stem cell transplantation for adolescent-/adult-onset cerebral form/cerebello-brainstem form of adrenoleukodystrophy.

引用

页数：14

共 50 条

[21] Autologous haematopoietic stem cell transplantation
Mancardi, GL
[J]. NEUROLOGICAL SCIENCES, 2005, 26 (Suppl 1) : S19 - S19
[22] Haematopoietic Stem Cell Transplantation for Lymphoma
Lie, A. K. W.
[J]. HONG KONG JOURNAL OF RADIOLOGY, 2011, 14 (04): : S69 - S75
[23] Adult haematopoietic stem cell niches
Genevieve M. Crane
Elise Jeffery
Sean J. Morrison
[J]. Nature Reviews Immunology, 2017, 17 : 573 - 590
[24] Haematopoietic stem cell transplantation in Tanzania
Mtenga, Janeth
Orf, Kate
Zheng, Jiexin
Chamba, Clara
Chuwa, Harrison
Luoga, Frederick
Malangahe, Stella W.
Iversen, Per Ole
Makani, Julie
[J]. BRITISH JOURNAL OF HAEMATOLOGY, 2021, 192 (01) : 17 - 21
[25] Editorial: haematopoietic stem cell transplantation
Jacobs, P
Wood, L
Sabit, R
[J]. TRANSFUSION SCIENCE, 2000, 23 (02): : 119 - 124
[26] Haematopoietic stem cell transplantation in children
R M Egeler
G Dini
[J]. Bone Marrow Transplantation, 2005, 35 : S1 - S1
[27] Haematopoietic stem cell transplantation as primary therapy of sporadic adult Burkitt lymphoma
Song, KW
Barnett, MJ
Gascoyne, RD
Horsman, DE
Forrest, DL
Hogge, DE
Lavoie, JC
Nantel, SH
Nevill, TJ
Shepherd, JD
Smith, CA
Sutherland, HJ
Voss, NJ
Toze, CL
Connors, JM
[J]. BRITISH JOURNAL OF HAEMATOLOGY, 2006, 133 (06) : 634 - 637
[28] Haematopoietic stem cell transplantation for cerebral X-linked adrenoleucodystrophy in adult
Paubelle, E.
Suarez, F.
Ghez, D.
Maillard, N.
Rubio, M.
Delarue, R.
Hirsch, I.
Varet, B.
Bellesmes, C.
Aubourg, P.
Buzyn, A.
[J]. BONE MARROW TRANSPLANTATION, 2009, 43 : S186 - S186
[29] Haematopoietic stem cell transplantation: indications, clinical developments and future directions
Boyiadzis, M
Pavletic, S
[J]. EXPERT OPINION ON PHARMACOTHERAPY, 2004, 5 (01) : 97 - 108
[30] Toxicity and efficacy of donor lymphocyte infusion after haematopoietic stem cell transplantation
Roncon, S
Bini-Antunes, M
Campilho, F
Barbosa, IL
Avila, A
Ferreira, S
Leal, H
Vaz, CP
Campos, A
Ferreira, RB
Pimentel, P
Carvalhais, A
[J]. BONE MARROW TRANSPLANTATION, 2006, 37 : S304 - S304

← 1 2 3 4 5 →