A phase I-II trial of autologous peripheral blood stem cell transplantation in the treatment of refractory autoimmune disease

H. Tsukamoto, K. Nagafuji, T. Horiuchi, T. Miyamoto, K. Aoki, K. Takase, H. Henzan, D. Himeji, T. Koyama, K. Miyake, Y. Inoue, H. Nakashima, T. Otsuka, Y. Tanaka, K. Nagasawa, M. Harada

研究成果: Contribution to journalArticle査読

59 被引用数 (Scopus)

抄録

Objectives: To carry out a phase I-II trial to elucidate the feasibility and efficacy of high dose cyclophosphamide (CY) supported by autologous peripheral blood stem cell transplantation (PBSCT) in the treatment of severe and refractory autoimmune disease (AD). Methods: Peripheral blood stem cells (PBSCs) were mobilised during haematological recovery after relatively high dose CY (2 g/m2) for 2 days, followed by administration of granulocyte colony stimulating factor. After collecting PBSCs-more than 2 × l0 6 CD34+ cells/kg-by apheresis, CD34+ cells were immunologically selected and cryopreserved. Eight patients were enrolled-five had systemic sclerosis (SSc) alone, one had SSc with systemic lupus erythematosus, one amyopathic dermatomyositis (ADM), and one Wegener's granulomatosis (WG). All of the patients were treated with high dose CY (50 mg/kg) for 4 days and autologous PBSCT. Results: Haematopoietic reconstitution was rapid and sustained. Toxicity due to the regimen included various infections such as pneumonia, sepsis, cystitis, herpes zoster, and acute heart failure. However, there was no treatment related mortality. Encouraging results were obtained after autologous PBSCT. Sclerosis of the skin was markedly improved in all of the patients with SSc. Interstitial pneumonia (IP), evaluated by PaO2, serum KL-6 levels, and pulmonary high resolution computed tomography, improved significantly. In a patient with ADM, severe and progressive IP also improved markedly. In a patient with WG, the size of the left orbital granuloma decreased substantially, resulting in reduction of the exophthalmos. Conclusions: These observations suggest that high dose CY with autologous PBSCT is feasible and may be effective in the treatment of severe and refractory AD.

本文言語英語
ページ(範囲)508-514
ページ数7
ジャーナルAnnals of the Rheumatic Diseases
65
4
DOI
出版ステータス出版済み - 4 2006

All Science Journal Classification (ASJC) codes

  • 免疫アレルギー学
  • リウマチ学
  • 免疫学
  • 生化学、遺伝学、分子生物学(全般)

フィンガープリント

「A phase I-II trial of autologous peripheral blood stem cell transplantation in the treatment of refractory autoimmune disease」の研究トピックを掘り下げます。これらがまとまってユニークなフィンガープリントを構成します。

引用スタイル