Hematopoietic stem cell transplantation (HCT) and cellular therapies including CAR-T and immune effector cells
Target Population
Patients with severe and refractory autoimmune diseases, especially multiple sclerosis
Care Setting
Specialized transplant and cellular therapy centers, including university hospitals and accredited transplant units
Key Highlights
Pioneered and led clinical research and trials on autologous HCT for severe multiple sclerosis, including the EBMT ASTIMS randomized phase II trial.
Held leadership roles in European Society for Blood and Marrow Transplantation (EBMT) and Joint Accreditation Committee (JACIE), advancing quality standards and accreditation in cellular therapies.
Promoted multidisciplinary collaboration across neurology, rheumatology, gastroenterology, immunology, hematology, and transplant communities to improve clinical outcomes.
Guideline-Based Recommendations
Diagnosis
Consider hematopoietic stem cell transplantation for patients with severe, refractory autoimmune diseases such as multiple sclerosis after multidisciplinary evaluation.
Management
Implement autologous HCT protocols in specialized centers with expertise in cellular therapies and immune reconstitution.
Incorporate quality assurance and risk-adapted benchmarking programs to monitor HCT outcomes as per FACT-JACIE accreditation standards.
Monitoring & Follow-up
Regularly assess clinical response and disability progression post-HCT to evaluate efficacy and adjust management.
Monitor immune reconstitution and potential complications following cellular therapies.
Risks
Recognize and manage risks associated with HCT and cellular therapies, including transplant-related morbidity and mortality.
Ensure adherence to regulatory and quality standards to minimize procedural risks.
Patient & Prescribing Data
Patients with severe and refractory autoimmune diseases, particularly multiple sclerosis, eligible for autologous HCT.
Autologous HCT has demonstrated efficacy in reducing disability and improving quality of life in refractory MS, supported by clinical trials and translational research.
Clinical Best Practices
Adopt a multidisciplinary approach involving neurologists, hematologists, immunologists, and other specialists for patient selection and management.
Maintain high standards of care through accreditation and quality improvement programs such as JACIE.
Engage in ongoing clinical research and trials to refine HCT protocols and expand indications for cellular therapies.
Provide patient-centered care with respect, dignity, and consistent availability.
