A Non-interventional Prospective Study on Allogeneic Stem Cell Transplantation in Patients With AL Amyloidosis

Light-chain (AL-) amyloidosis is a very rare monoclonal plasma cell disorder with poor prognosis. Rarity of disease has precluded performance of randomized controlled trials comparing various possible treatment modalities. In general, treatment of AL amyloidosis has been adapted from myeloma (MM) therapy. There is large experience with allo SCT in MM. Based on small series of patients and case reports allogeneic transplant has emerged as potentially effective. However, more formal proof of concept of using allogeneic hematopoietic transplantation for treatment of AL Amyloidosis is lacking.

Therefore, given the limitations of conventionally collected registry data (dubious follow-up information and extreme heterogeneity), we developed the: "EBMT non-interventional prospective study on allogeneic transplantation in AL Amyloidosis" which means that transplant centers that already do perform allogeneic transplants for AL Amyloidosis will be encouraged to register their patients with AL Amyloidosis very timely with the EBMT, followed by mandatory submission of EBMT MedB and follow-up forms. The diagnosis of AL Amyloidosis would be based on uniform criteria. All EBMT centres performing allogeneic transplants for Amyloidosis will be invited to participate in this study and centres will be asked to report all AL Amyloidosis cases referred for transplantation using a simple registration form and then to submit Amyloidosis MED B forms for each transplanted patient and follow-up forms as necessary. In conclusion, it should be possible to largely improve the usual quality of registry-based data and to generate scientifically sound knowledge on HSCT in an orphan disease such as AL Amyloidosis.