Assessment of Minimal Residual Disease (MRD) After Antineoplastic Treatment in Patients With AL Amyloidosis

In this study, the investigators seek to evaluate bone marrow and blood samples and treatment
responses to see if Minimal Residual Disease (MRD) (as described below), can be used as a
predictive method of response to treatment in amyloidosis.

Minimal residual disease (MRD) is a concept that has gained significant value as a prognostic
predictor and has become an emerging constituent of complete response (CR) reassessment in
multiple myeloma (MM) patients. Studies in MM have demonstrated that up to 30% of patients
achieving a CR after high-dose therapy will still have detectable MRD in the bone marrow as
measured by standard-sensitivity flow cytometry or by molecular assays. Virtually every study
examining MRD in MM has reported that among patients achieving a CR, those who were MRD
negative (MRD-) had a significantly superior progression-free survival, with some studies
reporting superior overall survival.

As amyloidosis is a disease that is very similar to multiple myeloma, the investigators wish
to evaluate the concept in this disease.

Inclusion Criteria:

- Biopsy-proven systemic AL amyloidosis defined as

- At least one + Congo Red stain

- Proof of a clonal plasma cell dyscrasia by:

- Immunofixation electrophoresis (IFE) of the urine or serum

- Light chain restriction based on Immunohistochemistry (IHC) in bone marrow plasma
cells or in the amyloid tissue

- Must be scheduled to undergo antineoplastic therapy (this may include high dose
melphalan and Autologous Stem Cell Transplantation) for AL Amyloidosis (Part II
enrollments only)

Exclusion Criteria:

- Co-existing Multiple Myeloma

- Prior antineoplastic treatment for AL amyloidosis at time of enrollment.

- Prior negative bone marrow biopsy showing no identifiable clone