Stem Cell Transplant Improves Severe Scleroderma Survival


Myeloablative autologous hematopoietic stem cell transplant may provide long-term benefits for patients with scleroderma.

Findings from a clinical trial suggest that transplanting a patient’s own blood-forming stem cells may be beneficial for patients with severe scleroderma. This treatment was found to improve survival and quality of life for patients with this serious autoimmune condition, according to a study published by The New England Journal of Medicine.

The investigational method includes chemotherapy and total body radiation to eradicate bone marrow, followed by a myeloablative autologous hematopoietic stem cell transplant (HSCT) to restart the immune system.

The National Institutes of Health (NIH)-funded study demonstrated that myeloablative HSCT was more effective than treatment with cyclophosphamide, an immunosuppressant.

Diffuse systemic sclerosis is a severe form of scleroderma that is often fatal and is characterized by hardening of the skin, connective tissues, and internal organs. Although patients are typically prescribed antirheumatic drugs and immunosuppressants to manage symptoms, these treatments have not been proven to provide long-term benefits, according to the NIH.

The safety and efficacy of stem cell transplant or cyclophosphamide was evaluated among 75 patients with diffuse systemic sclerosis who had lung or kidney involvement. Patients were randomized to receive HSCT or 1 year of cyclophosphamide.

During the myeloablative HSCT, physicians first collected blood-forming stem cells from patients before they underwent chemotherapy and radiation. The providers then infused the patients with their stem cells to reboot the immune system.

The researchers evaluated clinical outcomes, including death, survival without disease-related organ damage, progression of lung and skin disease, and quality of life.

At 4.5 years of follow-up, patients who underwent the transplant had significantly better clinical outcomes compared with cyclophosphamide-treated patients, according to the study.

The authors also found that 44% of patients treated with cyclophosphamide began antirheumatic therapy due to disease progression compared with 9% of transplant patients.

“We need effective therapies for scleroderma and other severe autoimmune diseases, which can be not only debilitating to the patient but also difficult to treat,” said Anthony S. Fauci, MD, director of the National Institute of Allergy and Infectious. “These results add to the growing evidence that stem cell transplants should be considered as a potential treatment option for people with poor-prognosis scleroderma.”

Notably, only 7 patients in the transplant cohort died during the study, compared with 14 in the drug therapy cohort, according to the study. Only 2 participants who received a transplant died as a result of disease progression compared with 11 deaths among patients who received cyclophosphamide, according to the study.

Another 2 deaths in the transplant arm were related to the therapy, but the authors said the rate is lower than previous statistics for HSCT. Additionally, no deaths were directly linked to treatment with cyclophosphamide, according to the study.

Overall, the authors found that transplantation offered long-term benefits compared with cyclophosphamide therapy; however, they discovered that myeloablative HSCT poses short-term risks, including infections and low blood cell counts.

The authors noted that infection rates were similar across cohorts, but more transplant patients developed varicella zoster infections.

The researchers said they plan to continue following these patients to determine long-term outcomes.

“Our findings indicate that undergoing stem cell transplantation for severe scleroderma poses more short-term risks but offers greater long-term gains than cyclophosphamide treatment,” said principal investigator Keith M. Sullivan, MD. “While treatment decisions should always be made on an individual basis, we hope that our work will help define a new standard of care for this severe, life-threatening autoimmune disease.”

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