Haematopoietic stem cell transplantation in severe autoimmune diseases – EBMT guidelines for physicians

by Alexey Bersenev on October 20, 2011 · 0 comments

in consensus, hematopoietic

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European Group for Blood and Marrow Transplantation (EBMT) has released a report about results of clinical trials for autoimmune diseases. This report summarizes all current data, collected from EBMT registry and provides a guidelines for physicians.

Since 1995, more than 3000 patients with severe drug-resistant autoimmune diseases (AD) underwent experimental hematopoietic stem cell transplantation (HSCT) around the world. EBMT’s report analyzes 1273 patients with such AD as multiple sclerosis, systemic sclerosis, lupus, rheumatoid arthritis, Crohn’s disease, type 1 diabetes and some others.

Some outcomes:

In the most recent general retrospective analysis, among 900 patients undergoing first autologous HSCT, the overall 5-year survival was 85%, with 43% PFS. Outcomes varied with diagnosis, age and centre activity. Retrospective analysis of limited numbers of allogeneic HSCT, including the use of donor T-cell infusions, confirmed the ability to achieve very prolonged remission, but with significantly more toxicity and TRM than with autologous HSCT.

Only when efficacy is formally demonstrated by controlled studies balancing the acute risks of HSCT vs the toxicity of chronic immunosuppressive treatment will HSCT be perceived as an alternative to, or reasonable escalation step after failure of standard treatment.

About necessity for guidelines:

For the present time, it is intended that these guidelines and recommendations will promote patient safety and facilitate harmonisation of procedural aspects, patient selection, data collection and coordination of prospective studies, with the aim of identifying the most appropriate clinical niche of HSCT in each AD, as well as supporting basic scientific research endeavours.

Full report in open access. Highly recommended!

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