Allo SCT in Amyloidosis Non-interventional Study
Condition: AL Amyloidosis
MeSH terms: Immunoglobulin Light-chain Amyloidosis; Amyloidosis
Study type: Observational [Patient Registry]

Summary: 
      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.
    

Detailed description: 
      Short description of the study:

      We plan to select those centres within the EBMT that in the past have performed any
      allogeneic transplant for amyloidosis and ask them to participate by completing a centre
      registration form. When they have a patient they want to include in this study, the centre
      will notify the CLWP data office of the planned allogeneic transplant the day before the
      transplant takes place. For this, the centre can use a patient registration form. Once a
      patient is included, MED B and regular follow-up are mandatory. Data entry of MED B forms and
      follow up will be performed in the way that is usual for the centre (either by the centre
      itself, by the national registry or by the Paris data office). The CLWP data office will keep
      track of the data submitted.

      Research design:

      This study is designed as a non-interventional prospective study.

      Study Population:

      Male or female subjects, 18 years to 60 years, with AL amyloidosis who will receive
      allogeneic transplantation.

      Data to be collected/analysed:

      The primary endpoint of this study is efficacy (best hematological remission and organ
      response) of allogeneic SCT in patients with AL amyloidosis. The secondary endpoints are
      acute and chronic GvHD, TRM and event-free and overall survival.

      Purpose of the non-interventional prospective study request:

      The main purpose of this non-interventional prospective study is the evaluation of the
      effectiveness of allogeneic transplant for AL Amyloidosis. It is planned that results will be
      presented on scientific symposia and that they will suffice for at least one publication
      (original article).
    

Observational model: Cohort
Time perspective: Prospective
Sampling method: Probability Sample
Study population: 
        Male or female subjects, 18 years to 60 years, with AL amyloidosis who will receive
        allogeneic transplantation
      
Keywords: AL Amyloidosis; Amyloidosis; allogeneic; HSCT