Leflunomide for Musculoskeletal GVHD After Allogeneic Stem Cell Transplant

• Status: Recruiting
• Trial end date: 2023-04-09
• Target enrollment: 0
• Participant gender: All

Summary

Graft versus host disease (GVHD) is a well-known complication of allogeneic transplant. In GVHD, the cells of the donor attack the patient's tissues and cause damage. It can affect any organ or system of the body. In a proportion of patients, it affects the joints and muscles. This is known as musculoskeletal GVHD. The standard treatment of musculoskeletal GVHD is steroids. However, these are usually needed for prolonged periods, and cause a large number of additional problems in transplant patients.

Leflunomide is a drug which has been used for several years in diseases like rheumatoid arthritis (RA). RA is an auto-immune disorder. The biological mechanisms underlying RA and musculoskeletal GVHD are quite similar. Hence it is likely that leflunomide may work in musculoskeletal GVHD also. The investigator have previously used leflunomide in a few patients with musculoskeletal GVHD and have found it to be extremely effective. Also, it was very safe (unlike steroids). Yet another advantage is that it is fairly cheap.

The purpose of the current study is to study the efficacy and safety of leflunomide in patients with musculoskeletal GVHD in a prospective way.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Tata Memorial Centre
• Treatments: Leflunomide

Criteria

Inclusion Criteria:

1. Willing to give written informed consent

2. Patients diagnosed with musculoskeletal mGvHD based on 2014 NIH consensus criteria (with diagnosis confirmed by biopsy only if clinically required).

3. Willing and able to comply with all study requirements, including treatment, and periodic assessments.

Exclusion Criteria:

1. Patients with known hypersensitivity to leflunomide especially previous Steven Johnson syndrome, toxic epidermal necrolysis after leflunomide.

2. Pregnant females

3. Patients with musculoskeletal manifestations explained by other potential causes ( (drugs, trauma, etc).

4. Patients with calculated glomerular filtration rate (GFR) <30ml/min at the time of screening.