Chronic GvHD continues to pose a major clinical challenge, accounting for significant morbidity and mortality post allogeneic haematopoietic stem cell transplant. Despite the success of multi-agent immunosuppressive therapies in reducing both the incidence and severity of acute GvHD, a similar improvement has not been seen in cGvHD. The incidence remains unchanged and the increase in the number of these transplants is reflected in the increase in the actual number of patients with cGvHD. The use of novel combinations of conventional, new approved and investigational agents have resulted in improved survival, but many patients continue to have poor clinical function. Consequently cGvHD must be considered one of the major clinical obstacles still facing the field of blood and marrow transplantation and there is an urgent need to evaluate alternative therapeutic strategies.

The EBMT is sponsoring a new clinical trial to evaluate the efficacy of the novel agent Myfortic® in combination with conventional agents, Cyclosporine A and Prednisone as 1st line treatment for extensive cGvHD. Entitled “ A randomised, double blinded, placebo controlled trial comparing Cyclosporine plus steroids with or without Myfortic® as primary treatment for extensive chronic graft versus host disease”; this study has been supported by a significant grant from Novartis. The objectives, treatment plan and evaluation schedule are detailed below.

The objectives of the study are:
Primary:

To improve the remission rates with the addition of Myfortic® to the standard therapy of Cyclosporine A and Prednisone

Secondary:

To spare the patients the long term use and side effects of corticosteroids
To decrease transplant related morbidity (infectious and non-infectious)
To study time to cessation of any immunosuppressive therapy
To decrease mortality at one year post transplant
To prospectively test the NIH severity index for cGvHD

Study Schema

Follow-up Schema

The trial is expected to start in April 2006 and centres will be selected on allo transplant graft activity.

Gérard Socié
LEWP Chair