Stem cell transplants for refractory autoimmune disease

Autologous peripheral blood stem cell transplantation (PBSCT) is effective for the treatment of severe and refractory autoimmune disease, according to a recent a phase I-II trial.The Japanese researchers, led by Dr Hiroshi Tsukamoto (Kyushu University Graduate School of Medicine), studied the use of high dose cyclophosphamide followed by PBSCT in eight patients: five with systemic sclerosis (SSc) alone, one with SSc plus systemic lupus erythematosus, one with amyopathic dermatomyositis (ADM), and one with Wegener's granulomatosis (WG).All of the patients were treated with high dose cyclophosphamide (50 mg/kg) for four days and autologous PBSCT. According to the researchers haematopoietic reconstitution was "rapid and sustained." However, a variety of toxic reactions including pneumonia, sepsis, cystitis, herpes zoster, acute heart failure, and treatment related mortality were identified. Tsukamoto's group report that skin sclerosis was "markedly improved" in all of the patients with systemic sclerosis, additionally, there was a significant improvement in cases of interstitial pneumonia. The authors note that for the patient with ADM, severe and progressive interstitial pneumonia improved markedly and in the patient with WG, the size of the left orbital granuloma decreased substantially.The researchers conclude that high-dose cyclophosphamide with autologous PBSCT "is feasible and may be effective in the treatment of severe and refractory autoimmune disease."Reference...