Adult Stem Cell Treatment of Scleroderma

Alan Tyndall; Daniel E. Furst


Curr Opin Rheumatol. 2007;19(6):604-610. 

In This Article

Prospective Randomized Trials

Figures 2 and 3 outline two ongoing studies: the ASTIS trial in Europe[17,18] and the SCOT trial in the USA. Both trials are similar in their selection criteria, primary outcome and control arms, but differ in the transplant regimen. ASTIS uses cyclophosphamide 200 mg/kg body weight and rabbit ATG, SCOT uses cyclophosphamide 120 mg/kg body weight, equine ATG and radiation 800 cGy (with shielding of the lungs to 200 cGy and renal shielding as well). The positive effect of cyclophosphamide alone (the control arm) on SSc was established in randomized placebo-controlled trials after initiation of both studies[19,20], confirming clinical practice and experience. Thus all patients in both trials receive active treatment.

The Autologous Stem cell Transplantation International Scleroderma (ASTIS) trial

The Scleroderma Cyclophosphamide Or Transplant (SCOT) trial

ASTIS is run under the auspices of the EBMT and EULAR and started randomizing 5 years ago. As of May 2007, 92 patients have been randomized, 44 to transplant and 48 to control. So far, no unexpected toxicity has been observed with one 'probable' transplant-related death occurring due to progressive cardiac failure. The independent safety committee adjudicated that no protocol violation had occurred and that there is no reason to change the protocol. It is planned to include a total of 120 patients.

The 226-patient SCOT trial is supported by the National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases and is currently recruiting. Twenty six patients have been randomized and 17 patients are undergoing testing for qualification. Thus far, there has been no mortality and there is no unexpected toxicity.

Both studies include extensive mechanistic studies relating to immune reconstitution, skin biopsy immunohistology, collagen and vascular remodelling, broncheoalveolar lavage cellular components and high-resolution computed tomography lung changes. An extensive biobank of serum, DNA and skin biopsy will facilitate further mechanistic studies, in particular, relating objective biomarkers to disease activity.


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