Stem Cell Transplant for Scleroderma

(SSc Trial)

This trial tests whether a combination of high-dose therapies, including cyclophosphamide (a chemotherapy drug), thiotepa (a chemotherapy agent), and total body irradiation (a form of radiation therapy), is safe and effective for people with systemic sclerosis, a condition that causes skin thickening and organ damage. The trial targets children, adolescents, and young adults who have not improved with at least two standard treatments and experience issues like worsening skin or lung problems, muscle inflammation, joint pain, or fingertip sores. Participants will undergo a stem cell transplant, using their own blood-forming cells to rebuild their immune system. As a Phase 2 trial, the research focuses on measuring how well the treatment works in an initial, smaller group of people.

The trial information does not specify if you need to stop taking your current medications. However, it mentions that participants must have failed to respond to at least two disease-modifying antirheumatic drugs (DMARDS) before joining, which might imply changes to your current treatment. Please consult with the trial coordinators for specific guidance.

Research has shown that using a person's own stem cells, known as autologous stem cell transplantation (ASCT), is a safe treatment option for people with systemic sclerosis (SSc). In past studies, patients who underwent this procedure found it both effective and manageable. One study examined different methods for preparing the stem cells and found that CD34-selected ASCT had a good safety record, with few serious side effects.

While side effects can occur with any medical treatment, evidence suggests that ASCT is generally safe for those with severe systemic sclerosis. The treatment uses a person's own stem cells to help repair and rebuild their immune system after a high-dose treatment that aims to eliminate the harmful immune cells causing the disease.

Researchers are excited about autologous stem cell transplantation for scleroderma because it offers a unique approach by using the patient's own stem cells to potentially reset the immune system. This method differs from standard treatments like immunosuppressive drugs, which mainly aim to reduce symptoms. The transplantation involves a conditioning regimen, including cyclophosphamide, thiotepa, and total body irradiation, to prepare the body for the stem cell infusion. By focusing on re-establishing a healthy immune response, this strategy may provide a more lasting solution than current options that mainly manage the disease rather than modifying its course.

Research has shown that using a person's own stem cells for treatment holds promise for severe systemic sclerosis (SSc). In one study, 87.5% of participants did not experience disease progression after one year, and 81.8% maintained this result after two years. Another study found that this treatment extended survival and symptom-free periods compared to other treatments. The risk of mortality from the transplant was relatively low, with only a 6% chance over six years. This evidence suggests that the treatment can be effective for individuals with severe forms of scleroderma.²³⁶⁷⁸