Outcome data from >10 000 multiple myeloma patients in the Danish and Swedish national registries.
multiple myeloma
national clinical databases
real-world data
Journal
European journal of haematology
ISSN: 1600-0609
Titre abrégé: Eur J Haematol
Pays: England
ID NLM: 8703985
Informations de publication
Date de publication:
Feb 2022
Feb 2022
Historique:
revised:
03
09
2021
received:
02
04
2021
accepted:
06
09
2021
pubmed:
14
9
2021
medline:
24
3
2022
entrez:
13
9
2021
Statut:
ppublish
Résumé
We describe real-world evidence (RWE) from the nationwide Swedish and Danish registries that provide important information on incidence and outcome in multiple myeloma (MM). First line treatment data on more than 10.000 MM patients from Denmark and Sweden between 2005-2018 are presented. Key results from research conducted within the Swedish and Danish myeloma registries are summarized, describing subgroups of patients with comorbidity, myeloma complications, and early relapse. We show that national guidelines, generated on results from randomized clinical trials (RCTs) are rapidly implemented and improve overall survival (OS). We find that both the incidence of MM and the median age at diagnosis is higher in national registries compared to results from referral centres, indicating a more complete coverage. This highlights the need of validation of prognostic scoring systems and indices in e.g., SMM and high-risk MM in a real- world-population. We show that these subgroups are unlikely to be captured in RCTs with narrow inclusion and exclusion criteria, that they have worse survival, and are in need of new treatment approaches. National registries that include all MM patients are an important source of knowledge on epidemiology, treatment and outcome with implications for the planning of MM care. Despite the introduction of new and better treatments, rapidly implemented in our countries, our registries uncover subgroups of patients that still have inferior outcome. Our RWE can help to identify important research questions to be studied in further clinical trials also in patients currently not included in RCTs.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
99-108Subventions
Organisme : Nordic Cancer Union
Organisme : Tömrermester Jörgen Holm og Hustru Elisa F. Hansens Mindelegat, Copenhagen, Denmark
Organisme : The Swedish State under the Agreement between the Swedish Government and the City Councils, the ALF- agreement ALFGBG-523261
Informations de copyright
© 2021 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.
Références
Turesson I, Bjorkholm M, Blimark CH, Kristinsson S, Velez R, Landgren O. Rapidly changing myeloma epidemiology in the general population: increased incidence, older patients, and longer survival. Eur J Haematol. 2018;101(2):237-244. 10.1111/ejh.13083
Facon T, Kumar S, Plesner T, et al. Daratumumab plus lenalidomide and dexamethasone for untreated myeloma. The New England journal of medicine. 2019;380(22):2104-2115.
Mateos MV, Cavo M, Blade J, et al. Overall survival with daratumumab, bortezomib, melphalan, and prednisone in newly diagnosed multiple myeloma (ALCYONE): a randomised, open-label, phase 3 trial. Lancet. 2020;395(10218):132-141.
Kumar SK, Dispenzieri A, Lacy MQ, et al. Continued improvement in survival in multiple myeloma: changes in early mortality and outcomes in older patients. Leukemia. 2014;28(5):1122-1128.
Costa LJ, Hari PN, Kumar SK. Differences between unselected patients and participants in multiple myeloma clinical trials in US: a threat to external validity. Leuk Lymphoma. 2016;57(12):2827-2832.
Shah JJ, Abonour R, Gasparetto C, et al. Analysis of common eligibility criteria of randomized controlled trials in newly diagnosed multiple myeloma patients and extrapolating outcomes. Clin lymphoma, Myeloma Leuk. 2017;17(9):575-83.e2.
Klausen TW, Gregersen H, Abildgaard N, et al. The majority of newly diagnosed myeloma patients do not fulfill the inclusion criteria in clinical phase III trials. Leukemia. 2019;33(2):546-549.
Ailawadhi S, Jagannath S, Narang M, et al. Connect MM registry as a national reference for United States multiple myeloma patients. Cancer Med. 2020;9(1):35-42.
Bergin K, Moore E, McQuilten Z, et al. Design and development of the Australian and New Zealand (ANZ) myeloma and related diseases registry. BMC Med Res Methodol. 2016;16(1):151.
Hájek R, Jarkovsky J, Maisnar V, et al. Real-world outcomes of multiple myeloma: retrospective analysis of the czech registry of monoclonal gammopathies. Clin Lymphoma, Myeloma Leuk. 2018;18(6):e219-e240.
Gimsing P, Holmström MO, Klausen TW, et al. The danish national multiple myeloma registry. Clin Epidemiol. 2016;8:583-587.
Blimark CH, Turesson I, Genell A, et al. Outcome and survival of myeloma patients diagnosed 2008-2015. real-world data on 4904 patients from the swedish myeloma registry. Haematologica. 2018;103(3):506-513.
Blimark C, Nahi H, Izarra A, et al. Myeloma. Kvalitetsregisterrapport. 2020;7. ISBN 978-91-87663-25-3.
Hansson MSR, Hareth N, Carlson K, Lauri B, Blimark C. Nationellt Vårdprogram Myelom. 2020. https://kunskapsbankencancercentrumse/diagnoser/myelom/vardprogram/
(DMSG) DMSG. Diagnostik og Behandling af Myelomatose Retningslinje 2017. 2017. https://myelomahematologydk/indexphp/vejledninger-dmsg
Szabo AG, Klausen TW, Abildgaard N, et al. Incidence and clinical characteristics of multiple myeloma with low M-protein levels and normal values of hemoglobin, creatinine, calcium, and serum free light chain ratio. Blood Cancer J. 2021;11(4):70.
Sørrig R, Klausen TW, Salomo M, et al. Smoldering multiple myeloma risk factors for progression: a Danish population-based cohort study. Eur J Haematol. 2016;97(3):303-309.
NftDsg A. Dansk Myelomatose Database Årsrapport 2019. 2020.
Sneyd MJ, Cox B, Morison IM. Trends in myeloma incidence, mortality and survival in New Zealand (1985-2016). Cancer Epidemiol. 2019;60:55-59.
Mateos M-V, Hernández M-T, Giraldo P, et al. Lenalidomide plus dexamethasone for high-risk smoldering multiple myeloma. N Engl J Med. 2013;369(5):438-447.
Pérez-Persona E, Vidriales M-B, Mateo G, et al. New criteria to identify risk of progression in monoclonal gammopathy of uncertain significance and smoldering multiple myeloma based on multiparameter flow cytometry analysis of bone marrow plasma cells. Blood. 2007;110(7):2586-2592.
Kyle RA, Rajkumar SV. Monoclonal gammopathy of undetermined significance and smouldering multiple myeloma: emphasis on risk factors for progression. Br J Haematol. 2007;139(5):730-743.
Kyle RA, Remstein ED, Therneau TM, et al. Clinical course and prognosis of smoldering (asymptomatic) multiple myeloma. N Engl J Med. 2007;356(25):2582-2590.
Kristinsson SY, Holmberg E, Blimark C. Treatment for high-risk smoldering myeloma. N Engl J Med. 2013;369(18):1762-1763.
Group IMW. Criteria for the classification of monoclonal gammopathies, multiple myeloma and related disorders: a report of the International myeloma working group. Br J Haematol. 2003;121(5):749-757.
Mateos M-V, Kumar S, Dimopoulos MA, et al. International myeloma working group risk stratification model for smoldering multiple myeloma (SMM). Blood Cancer J. 2020;10(10):102.
Dimopoulos MA, Moulopoulos LA, Maniatis A, Alexanian R. Solitary plasmacytoma of bone and asymptomatic multiple myeloma. Blood. 2000;96(6):2037-2044.
Nahi H, Genell A, Wålinder G, et al. Incidence, characteristics, and outcome of solitary plasmacytoma and plasma cell leukemia. population-based data from the swedish myeloma register. Eur J Haematol. 2017;99(3):216-222.
Wålinder G, Samuelsson J, Näsman P, et al. Outcome and characteristics of non-measurable myeloma: a cohort study with population-based data from the swedish myeloma registry. Eur J Haematol. 2020;104(5):376-382. 10.1111/ejh.13351
Kyle RA, Gertz MA, Witzig TE, et al. Review of 1027 patients with newly diagnosed multiple myeloma. Mayo Clin Proc. 2003;78(1):21-33.
Chawla SS, Kumar SK, Dispenzieri A, et al. Clinical course and prognosis of non-secretory multiple myeloma. Eur J Haematol. 2015;95(1):57-64.
Holmström MO, Gimsing P, Abildgaard N, et al. Causes of early death in multiple myeloma patients who are ineligible for high-dose therapy with hematopoietic stem cell support: a study based on the nationwide danish myeloma database. Am J Hematol. 2015;90(4):E73-E74.
Thidemann Andersen K, Klausen T, Abildgaard N, et al. Causes of early death in multiple myeloma patients treated with high-dose therapy followed by autologous stem cell transplantation: a study based on the nationwide danish multiple myeloma registry. Am J Hematol. 2017;92(10):E611-E614.
Augustson BM, Begum G, Dunn JA, et al. Early mortality after diagnosis of multiple myeloma: analysis of patients entered onto the United kingdom medical research council trials between 1980 and 2002-medical research council adult leukaemia working party. J Clin Oncol. 2005;23(36):9219-9226.
Moreau P, Cavo M, Sonneveld P, et al. Combination of international scoring system 3, high lactate dehydrogenase, and t(4;14) and/or del(17p) identifies patients with multiple myeloma (MM) treated with front-line autologous stem-cell transplantation at high risk of early MM progression-related death. J Clin Oncol. 2014;32(20):2173-2180.
Vangsted AJ, Klausen TW, Andersen NF, et al. Improved survival of multiple myeloma patients with late relapse after high-dose treatment and stem cell support, a population-based study of 348 patients in Denmark in 1994-2004. Eur J Haematol. 2010;85(3):209-216.
Kumar S, Mahmood ST, Lacy MQ, et al. Impact of early relapse after auto-SCT for multiple myeloma. Bone Marrow Transplant. 2008;42(6):413-420.
Helm-Petersen S, Sorrig R, Klausen TW, et al. Early relapsed disease of multiple myeloma following up-front HDM-ASCT: a study based on the danish multiple myeloma registry in the period 2005 to 2014. Leukemia. 2018;32(9):2054-2057.
Majithia N, Rajkumar SV, Lacy MQ, et al. Early relapse following initial therapy for multiple myeloma predicts poor outcomes in the era of novel agents. Leukemia. 2016;30(11):2208-2213.
Corre J, Montes L, Martin E, et al. Early relapse after autologous transplant for myeloma is associated with poor survival regardless of cytogenetic risk. Haematologica. 2020;105(9):e480-e483.
Szabo AG, Thorsen J, Iversen KF, et al. Clinically-suspected cast nephropathy: a retrospective, national, real-world study. Am J Hematol. 2020;95(11):1352-1360.
Palumbo A, Rajkumar SV, San Miguel JF, et al. International Myeloma Working Group consensus statement for the management, treatment, and supportive care of patients with myeloma not eligible for standard autologous stem-cell transplantation. J Clin Oncol. 2014;32(6):587-600.
Palumbo A, Bringhen S, Mateos MV, et al. Geriatric assessment predicts survival and toxicities in elderly myeloma patients: an International myeloma working group report. Blood. 2015;125(13):2068-2074.
Gregersen H, Vangsted AJ, Abildgaard N, et al. The impact of comorbidity on mortality in multiple myeloma: a danish nationwide population-based study. Cancer Med. 2017;6(7):1807-1816.
Cook G, Royle KL, Pawlyn C, et al. A clinical prediction model for outcome and therapy delivery in transplant-ineligible patients with myeloma (UK myeloma research alliance risk profile): a development and validation study. Lancet Haematol. 2019;6(3):e154-e166.
Redder L, Klausen TW, Vangsted AJ, et al. Validation of a new clinical prediction model for outcome in newly diagnosed multiple myeloma patients not eligible for autologous stem-cell transplantation; a population-based study from the danish national multiple myeloma registry. Br J Haematol. 2021;193(1):119-124. In Press. 2020.
Richardson PG, San Miguel JF, Moreau P, et al. Interpreting clinical trial data in multiple myeloma: translating findings to the real-world setting. Blood Cancer J. 2018;8(11):109.
Chari A, Romanus D, Palumbo A, et al. Randomized clinical trial representativeness and outcomes in real-world patients: comparison of 6 hallmark randomized clinical trials of relapsed/refractory multiple myeloma. Clin lymphoma Myeloma Leuk. 2020;20(1):8-17.
Redder L, Klausen TW, Vangsted AJ, et al. Validation of a new clinical prediction model for outcome in newly diagnosed multiple myeloma patients not eligible for autologous stem-cell transplantation; a population-based study from the danish national multiple myeloma registry. Br J Haematol. 2021;193(1):119-124. Submitted. 2020.
Myelomaregistry S. INCA Patientöversikt, Swedish Myeloma Registry. 2020. https://cancercentrumse/samverkan/cancerdiagnoser/blod-lymfom-myelom/myelom/patientoversikt/
Sørrig R, Klausen TW, Salomo M, Vangsted A, Gimsing P. Risk factors for blood stream infections in multiple myeloma: a population-based study of 1154 patients in denmark. Eur J Haematol. 2018;101(1):21-27.
Sørrig R, Klausen TW, Salomo M, et al. Immunoparesis in newly diagnosed multiple myeloma patients: effects on overall survival and progression free survival in the danish population. PLOS ONE. 2017;12(12):e0188988.