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High-dose immunoablative therapy
High-dose immunoablative therapy uses intensive chemotherapy and/or radiation to eliminate the patient's immune system, followed by autologous hematopoietic stem cell transplantation to reconstitute a new, disease-tolerant immune system.
High-dose immunoablative therapy uses intensive chemotherapy and/or radiation to eliminate the patient's immune system, followed by autologous hematopoietic stem cell transplantation to reconstitute a new, disease-tolerant immune system. Used for Severe systemic sclerosis (scleroderma), Severe refractory systemic lupus erythematosus, Severe refractory rheumatoid arthritis.
At a glance
| Generic name | High-dose immunoablative therapy |
|---|---|
| Sponsor | National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) |
| Drug class | Immunoablative therapy with autologous hematopoietic stem cell transplantation |
| Modality | Small molecule |
| Therapeutic area | Immunology / Rheumatology |
| Phase | Phase 3 |
Mechanism of action
This approach aims to 'reset' the immune system by destroying autoreactive T and B cells responsible for autoimmune disease, then restoring immune function with reinfused patient stem cells that have been selected or manipulated to prevent disease recurrence. The goal is to achieve long-term remission or cure of severe autoimmune conditions by eliminating the pathogenic immune clone while preserving protective immunity.
Approved indications
- Severe systemic sclerosis (scleroderma)
- Severe refractory systemic lupus erythematosus
- Severe refractory rheumatoid arthritis
Common side effects
- Infection (bacterial, viral, fungal)
- Mucositis
- Hematologic toxicity (neutropenia, thrombocytopenia)
- Organ toxicity (cardiac, renal, hepatic)
- Graft-versus-host disease (GVHD)
- Secondary malignancy
Key clinical trials
- Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS) (PHASE3)
- AHSCT With Fludarabine and Cyclophosphamide Based Conditioning Regimes in Patients With Multiple Sclerosis (PHASE1)
- Campath-1h Phase I/II Pilot Trial as Immunoablative Therapy for Refractory Systemic Sclerosis
- High-Dose Intravenous (IV) Cyclophosphamide Versus Monthly IV Cyclophosphamide (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
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