By Patrick Hayden, consultant haematologist and clinical lead for the myeloma service at St James’s Hospital, Dublin, Ireland. EBMT Chronic Malignancies Working Party, PCD Subcommittee Chair.
The Chronic Malignancies Working Party of the EBMT (CMWP-EBMT) joins the Myeloma global community this Friday 27th September, to mark European Myeloma Day. This year, the focus is on clinical trials and the challenges surrounding access to them.
The CMWP Plasma Cell Diseases (PCD) subcommittee has long been focused on improving the outcomes of patients with myeloma. There have been many new treatments introduced over the last quarter century: proteasome inhibitors like bortezomib, immunomodulatory drugs like lenalidomide and pomalidomide and monoclonal antibodies like Daratumumab and Isatuximab. More recently, we have entered the era of immunotherapy and are seeing unprecedented responses to CAR T therapies like Idacel and Ciltacel and bispecific antibodies like Teclistamab, Talquetamab and Elranatamab. We only have access to these life-changing therapies because patients and their relatives were willing to become involved in the randomised clinical trials which led to their regulatory approval. As a result of these efforts, the median survival in younger transplant-eligible patients is now approaching ten years and myeloma has become an excellent example of cancer as a chronic disease.
Clinical trials require the involvement of professionals from several fields: nurses, data managers, pharmacists, and doctors, among others. These trials can only be done in centres that develop and fund clinical trial infrastructure and where ongoing funding of staff is assured. In myeloma, the evidence for the benefit of autologous transplantation in myeloma was developed by key academic cooperative trials groups in France (IFM) and in the UK (MRC). More recently, commercially sponsored trials such as the DETERMINATION and FORTE clinical trials have supported the continuing role of high dose melphalan with peripheral blood stem cell support in the treatment of younger, fitter patients with myeloma.
The CMWP PCD subcommittee studies continue to provide insight into changing treatment trends in myeloma. In the last year alone, there have been several novel observations:
Remi Tilmont and colleagues reported that carfilzomib, lenalidomide and dexamethasone followed by a second autologous transplant is an effective strategy following a first relapse in myeloma (https://pubmed.ncbi.nlm.nih.gov/37543712/);
Meral Beksac led a study which found that intensification of melphalan-based conditioning with bortezomib does not improve survival outcomes in newly diagnosed myeloma (https://pubmed.ncbi.nlm.nih.gov/38297040/);
Laurent Garderet showed that patients can achieve promising long-term outcomes and renal responses following autologous hematopoietic stem cell transplantation for the rare plasma cell disorder, light chain deposition disease (https://pubmed.ncbi.nlm.nih.gov/38546696/).
Patients with myeloma can now generally look forward to many years in good health. The CMWP-EBMT, alongside national and international cooperative groups, continues to promoting research aimed at improving the quality of life of myeloma patients. On the 27th of September, we join with others in celebrating all those patients and professionals involved in clinical trials. Between us, we look forward to further improving the lives of all patients with myeloma.