EMN 2021 | Early diagnosis and optimal treatment of AL amyloidosis
Paolo Milani, MD, PhD, Amyloidosis Research and Treatment Center, Pavia, Italy, gives a summary of his talk on the importance of early diagnosis and the optimal treatment of immunoglobin light-chain (AL) amyloidosis. Dr Milani reports that early diagnosis is crucial for improving the survival of patients with AL amyloidosis, as well as outlining the importance of cardiac and renal biomarkers. These biomarkers are also important for risk-stratification and therapy optimization. Patients with early-stage disease are treated with autologous stem cell transplantation. For intermediate-risk patients who are transplant-ineligible, a Phase III trial (NCT01078454) investigating bortezomib, melphalan and dexamethasone in combination has demonstrated efficacy. For intermediate-risk patients who are transplant-eligible, the ANDROMEDA trial (NCT03201965) evaluating daratumumab in combination with bortezomib, cyclophosphamide and dexamethasone has also shown promising results. Treatment of high-risk patients with AL amyloidosis remains an unmet need. For relapsed/refractory disease, Dr Milani highlights the use of daratumumab, lenalidomide and pomalidomide. This interview took place during the 2021 European Myeloma Network (EMN) congress.
Transcript (edited for clarity)
I’d like to thank again the EMN Committee organization for having a talk, had a couple of talk about amyloidosis at these important meeting. And I discuss in the meeting the importance of early diagnosis and the optimal therapy for patients with this disease. Early diagnosis is fundamental, because patients with amyloidosis that were diagnosed in the early stage of the disease have more and a better survival compared to others and cardiac biomarkers are fundamental for the assessment of diagnosis...
I’d like to thank again the EMN Committee organization for having a talk, had a couple of talk about amyloidosis at these important meeting. And I discuss in the meeting the importance of early diagnosis and the optimal therapy for patients with this disease. Early diagnosis is fundamental, because patients with amyloidosis that were diagnosed in the early stage of the disease have more and a better survival compared to others and cardiac biomarkers are fundamental for the assessment of diagnosis.
Of particular importance for those patients in, with monoclonal gammopathy of undetermined significance, in which it is fundamental to check for cardiac and renal biomarkers to have the suspect and then the diagnosis of AL amyloidosis. Cardiac biomarkers are also fundamental for the optimal therapy.
And we discuss in the talk, the different treatment regimens for the different populations. So, those in the so called low-risk patient disease, so those are with low cardiac biomarkers with a mild cardiac involvement, those in an intermediate-risk disease and those in the so-called high-risk population. For all the different three groups we had the different approaches, that move from autologous stem cell transplant for those in the early stage of the disease and for the intermediate group we have now two Phase III studies that reported the optimal therapy for these subgroup. So, the bortezomib, melphalan and dexamethasone for the transplant-ineligible and of course in the use of daratumumab in combination with bortezomib for all those eligible to transplant. And thanks to the ANDROMEDA study results, probably these will be the new gold standard for newly diagnosed AL patients.
And lastly the so-called high-risk patients population, indeed there’s still an unmet need. And in these subgroup of patients, an approach with subsequent therapy or with the use of a low-dose treatment is the best option so far. Also, in this subset, try to enrich the best hematologic response, a rapid and deep hematologic decrease of the free light chain, translates in better overall survival. Of course, also for those patients, we have to treat and evaluate response quickly and maybe change treatment strategy quickly in order to try to obtain a profound reduction of the free light chain.
Lastly for the optimal therapy, we have now several options for the relapsed/refractory disease. Again, daratumumab is an important option if available, among patients that failed first-line therapy. Immunomodulatory agents represent a backbone of therapy so far, with lenalidomide and pomalidomide that demonstrate an efficacy in several studies. And lastly for those who are refractory also to daratumumab or to immunomodulatory agents, we have other options, and also we have the importance of enrolment in clinical trials and collaboration among centers and among experts is fundamental in particular for rare disease such as AL amyloidosis.
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