Sclerosis: Autologous Cell Transfers May Help Patients

Jennifer Garcia

August 17, 2014

Injections of autologous stromal vascular fraction (SVF) may improve hand function and decrease pain among patients with systemic sclerosis (SSc), according to a new study published online August 11 in the Annals of the Rheumatic Diseases.

The phase 1, open-label study enrolled 12 female patients with SSc and a Cochin Hand Function Scale score higher than 20/90. Patients receiving vasodilator or immunosuppressive therapy in the 3 months before or 6 months after enrollment were excluded from the study. Patients were administered subcutaneous injections of autologous SVF into each finger of both hands and were evaluated over the course of a 6-month period.

The researchers, led by Brigitte Granel, MD, and Aurélie Daumas, MD, from the Assistance Publique Hôpitaux de Marseilles, France, found that SVF injection resulted in a decrease of 53.7% from baseline in the Raynaud's Condition Score by month 2 (P < .001) and 67.5% by month 6 (P < .001). By month 2, the researchers saw a 47.4% decrease from baseline in the Cochin Hand Function Scale score; they saw a 56.0% decrease by month 6 (P < .001 for both). In addition, patients experienced a 37.8% reduction in hand pain by 2 months (P = .001) and a 41.7% reduction by 6 months (P < .001).

"To our knowledge, SVF is the first treatment improving such hand manifestations and with favorable impact on quality of life in patients with SSc," the study authors write.

Asked by Medscape Medical News about other similar approaches being used to treat patients with SSc, Dr. Daumas noted that hematopoietic stem cell transplants have been evaluated in this setting.

"While there has been some success with this approach, it is quite toxic, and so is generally restricted to patients with more severe forms of scleroderma. The approach we used in this study is very benign, with no chemotherapy and very few side effects," Dr. Daumas said.

The authors note that SVF injection was the only therapeutic change during the study period, as other treatments and physiotherapy schedules were not altered in the 3 months preceding or the 6 months after the procedure.

In addition to improvements in the Raynaud's Condition Score and Cochin Hand Function Scale score, patients reported a significant improvement in their general health status by month 2, according to the Scleroderma Health Assessment Questionnaire (P = .001), which was sustained throughout the 6-month follow-up period.

"Women who were previously unable to apply make-up or cut their children¹s food for them, or [were] otherwise unable to do simple tasks of daily life, report that they are now able to do these tasks with ease," and "[t]he study carefully quantified these changes and others with validated clinical tools and demonstrated statistical and clinical significance in a number of parameters," Dr. Daumas said.

The study authors acknowledge that although the results are encouraging, these findings are preliminary and will require confirmation in larger clinical trials.

"Of course, we recognize that this was an open-label study with no controls," concluded Dr. Daumas."[I]t needs to be followed by a properly controlled study with blinded assessments and the other tools of rigorous clinical science to determine the ultimate significance of these findings to patients with scleroderma."

When asked to comment on these findings, Dinesh Khanna, MD, and Jennifer Waljee, MD, from the University of Michigan Scleroderma Program and University of Michigan Hand Center, Ann Arbor, respectively, noted: "The study population included long-standing disease duration with moderate-to-significant impairment in hand function. [The m]ajority of current trials are focused on early skin and lung involvement and do not address this unmet need. Therefore, this approach is novel and has potential to have significant impact in patients with scleroderma."

The study authors reported minor adverse events associated with the procedure, such as transient paresthesia and postinjection pain, which spontaneously resolved. Dr. Dinesh and Dr. Waljee also note the possibility of damage to the palmar structures, increased compartment pressure, or acute carpal tunnel syndrome.

"Although rare in studies with small sample size, the true incidence and risk factors for these adverse effects is not yet known," note Dr. Dinesh and Dr. Waljee. "There are no effective treatments for scleroderma-associated hand contractors, and this therapy will provide an effective therapy, if shown to be superior and safe in a large [randomized controlled trial]," they concluded.

Funding for this study was provided by the Groupe Francophone de Recherche sur la Sclérodermie. The authors have disclosed no relevant financial relationships. Dr. Khanna has served as consultant and/or received grant support from Actelion, Bayer, Biogen-Idec, BMS, Cytori, DIGNA, Genentech/Roche, Gilead, InterMune, Lycera, Merck, and sanofi-aventis. Dr. Waljee serves as a consultant for 3M Health Systems.

Ann Rheum Dis. Published online August 11, 2014. Full text


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