Multiple Myeloma Treatment in Real-world Clinical Practice: Results of a Prospective, Multinational, Noninterventional Study

EMMOS Investigators

Research output: Contribution to journalArticle

Abstract

Multiple myeloma (MM) remains an incurable disease, with little information available on its management in real-world clinical practice. The results of the present prospective, noninterventional observational study revealed great diversity in the treatment regimens used to treat MM. Our results also provide data to inform health economic, pharmacoepidemiologic, and outcomes research, providing a framework for the design of protocols to improve the outcomes of patients with MM. Background: The present prospective, multinational, noninterventional study aimed to document and describe real-world treatment regimens and disease progression in multiple myeloma (MM) patients. Patients and Methods: Adult patients initiating any new MM therapy from October 2010 to October 2012 were eligible. A multistage patient/site recruitment model was applied to minimize the selection bias; enrollment was stratified by country, region, and practice type. The patient medical and disease features, treatment history, and remission status were recorded at baseline, and prospective data on treatment, efficacy, and safety were collected electronically every 3 months. Results: A total of 2358 patients were enrolled. Of these patients, 775 and 1583 did and did not undergo stem cell transplantation (SCT) at any time during treatment, respectively. Of the patients in the SCT and non-SCT groups, 49%, 21%, 14%, and 15% and 57%, 20%, 12% and 10% were enrolled at treatment line 1, 2, 3, and ≥ 4, respectively. In the SCT and non-SCT groups, 45% and 54% of the patients had received bortezomib-based therapy without thalidomide/lenalidomide, 12% and 18% had received thalidomide/lenalidomide-based therapy without bortezomib, and 30% and 4% had received bortezomib plus thalidomide/lenalidomide-based therapy as frontline treatment, respectively. The corresponding proportions of SCT and non-SCT patients in lines 2, 3, and ≥ 4 were 45% and 37%, 30% and 37%, and 12% and 3%, 33% and 27%, 35% and 32%, and 8% and 2%, and 27% and 27%, 27% and 23%, and 6% and 4%, respectively. In the SCT and non-SCT patients, the overall response rate was 86% to 97% and 64% to 85% in line 1, 74% to 78% and 59% to 68% in line 2, 55% to 83% and 48% to 60% in line 3, and 49% to 65% and 36% and 45% in line 4, respectively, for regimens that included bortezomib and/or thalidomide/lenalidomide. Conclusion: The results of our prospective study have revealed great diversity in the treatment regimens used to manage MM in real-life practice. This diversity was linked to factors such as novel agent accessibility and evolving treatment recommendations. Our results provide insight into associated clinical benefits.

Original languageEnglish
JournalClinical Lymphoma, Myeloma and Leukemia
DOIs
Publication statusAccepted/In press - Jan 1 2018

Fingerprint

Multiple Myeloma
Stem Cell Transplantation
Thalidomide
Cell Transplantation
Therapeutics
Selection Bias
Cerebral Palsy
Patient Selection
Observational Studies
Disease Progression
History
Economics
Outcome Assessment (Health Care)
Prospective Studies
Safety

Keywords

  • Bortezomib
  • Global
  • Observational study
  • Routine practice
  • Stem cell transplantation

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

Cite this

@article{4e45bf22dfab4f63a3879a8b02c36250,
title = "Multiple Myeloma Treatment in Real-world Clinical Practice: Results of a Prospective, Multinational, Noninterventional Study",
abstract = "Multiple myeloma (MM) remains an incurable disease, with little information available on its management in real-world clinical practice. The results of the present prospective, noninterventional observational study revealed great diversity in the treatment regimens used to treat MM. Our results also provide data to inform health economic, pharmacoepidemiologic, and outcomes research, providing a framework for the design of protocols to improve the outcomes of patients with MM. Background: The present prospective, multinational, noninterventional study aimed to document and describe real-world treatment regimens and disease progression in multiple myeloma (MM) patients. Patients and Methods: Adult patients initiating any new MM therapy from October 2010 to October 2012 were eligible. A multistage patient/site recruitment model was applied to minimize the selection bias; enrollment was stratified by country, region, and practice type. The patient medical and disease features, treatment history, and remission status were recorded at baseline, and prospective data on treatment, efficacy, and safety were collected electronically every 3 months. Results: A total of 2358 patients were enrolled. Of these patients, 775 and 1583 did and did not undergo stem cell transplantation (SCT) at any time during treatment, respectively. Of the patients in the SCT and non-SCT groups, 49{\%}, 21{\%}, 14{\%}, and 15{\%} and 57{\%}, 20{\%}, 12{\%} and 10{\%} were enrolled at treatment line 1, 2, 3, and ≥ 4, respectively. In the SCT and non-SCT groups, 45{\%} and 54{\%} of the patients had received bortezomib-based therapy without thalidomide/lenalidomide, 12{\%} and 18{\%} had received thalidomide/lenalidomide-based therapy without bortezomib, and 30{\%} and 4{\%} had received bortezomib plus thalidomide/lenalidomide-based therapy as frontline treatment, respectively. The corresponding proportions of SCT and non-SCT patients in lines 2, 3, and ≥ 4 were 45{\%} and 37{\%}, 30{\%} and 37{\%}, and 12{\%} and 3{\%}, 33{\%} and 27{\%}, 35{\%} and 32{\%}, and 8{\%} and 2{\%}, and 27{\%} and 27{\%}, 27{\%} and 23{\%}, and 6{\%} and 4{\%}, respectively. In the SCT and non-SCT patients, the overall response rate was 86{\%} to 97{\%} and 64{\%} to 85{\%} in line 1, 74{\%} to 78{\%} and 59{\%} to 68{\%} in line 2, 55{\%} to 83{\%} and 48{\%} to 60{\%} in line 3, and 49{\%} to 65{\%} and 36{\%} and 45{\%} in line 4, respectively, for regimens that included bortezomib and/or thalidomide/lenalidomide. Conclusion: The results of our prospective study have revealed great diversity in the treatment regimens used to manage MM in real-life practice. This diversity was linked to factors such as novel agent accessibility and evolving treatment recommendations. Our results provide insight into associated clinical benefits.",
keywords = "Bortezomib, Global, Observational study, Routine practice, Stem cell transplantation",
author = "{EMMOS Investigators} and Mohamad Mohty and Evangelos Terpos and Mateos, {Maria Victoria} and Michele Cavo and Sandra Lejniece and Meral Beksac and Bekadja, {Mohamed Amine} and Wojciech Legiec and Meletios Dimopoulos and Svetlana Stankovic and Dur{\'a}n, {Maria Soledad} and {De Stefano}, Valerio and Alessandro Corso and Yulia Kochkareva and Edward Laane and Christian Berthou and Hans Salwender and Zvenyslava Masliak and Valdas Pečeliūnas and Wolfgang Willenbacher and Jo{\~a}o Silva and Vernon Louw and Damir Nemet and Zita Borb{\'e}nyi and Uri Abadi and Pedersen, {Robert Schou} and Peter Černelč and Anna Potamianou and Catherine Couturier and Caroline Feys and Florence Thoret-Bauchet and Mario Boccadoro and Mohamed Bekadja and Hamladji, {Rose Marie} and Ali, {Hocine Ait} and Selma Hamdi and Hadj Touhami and Mansour, {Nourredine Sidi} and Wolfgang Willenbacher and Werner Linkesch and Damir Nemet and Pedersen, {Robert Shou} and Niels Abildgaard and Edward Laane and Marju Hein and Alessandro Corso and Emanuele Angelucci and Attilio Guarini and Atelda Romano and Marco Gobbi",
year = "2018",
month = "1",
day = "1",
doi = "10.1016/j.clml.2018.06.018",
language = "English",
journal = "Clinical Lymphoma, Myeloma and Leukemia",
issn = "2152-2669",
publisher = "Cancer Media Group",

}

TY - JOUR

T1 - Multiple Myeloma Treatment in Real-world Clinical Practice

T2 - Results of a Prospective, Multinational, Noninterventional Study

AU - EMMOS Investigators

AU - Mohty, Mohamad

AU - Terpos, Evangelos

AU - Mateos, Maria Victoria

AU - Cavo, Michele

AU - Lejniece, Sandra

AU - Beksac, Meral

AU - Bekadja, Mohamed Amine

AU - Legiec, Wojciech

AU - Dimopoulos, Meletios

AU - Stankovic, Svetlana

AU - Durán, Maria Soledad

AU - De Stefano, Valerio

AU - Corso, Alessandro

AU - Kochkareva, Yulia

AU - Laane, Edward

AU - Berthou, Christian

AU - Salwender, Hans

AU - Masliak, Zvenyslava

AU - Pečeliūnas, Valdas

AU - Willenbacher, Wolfgang

AU - Silva, João

AU - Louw, Vernon

AU - Nemet, Damir

AU - Borbényi, Zita

AU - Abadi, Uri

AU - Pedersen, Robert Schou

AU - Černelč, Peter

AU - Potamianou, Anna

AU - Couturier, Catherine

AU - Feys, Caroline

AU - Thoret-Bauchet, Florence

AU - Boccadoro, Mario

AU - Bekadja, Mohamed

AU - Hamladji, Rose Marie

AU - Ali, Hocine Ait

AU - Hamdi, Selma

AU - Touhami, Hadj

AU - Mansour, Nourredine Sidi

AU - Willenbacher, Wolfgang

AU - Linkesch, Werner

AU - Nemet, Damir

AU - Pedersen, Robert Shou

AU - Abildgaard, Niels

AU - Laane, Edward

AU - Hein, Marju

AU - Corso, Alessandro

AU - Angelucci, Emanuele

AU - Guarini, Attilio

AU - Romano, Atelda

AU - Gobbi, Marco

PY - 2018/1/1

Y1 - 2018/1/1

N2 - Multiple myeloma (MM) remains an incurable disease, with little information available on its management in real-world clinical practice. The results of the present prospective, noninterventional observational study revealed great diversity in the treatment regimens used to treat MM. Our results also provide data to inform health economic, pharmacoepidemiologic, and outcomes research, providing a framework for the design of protocols to improve the outcomes of patients with MM. Background: The present prospective, multinational, noninterventional study aimed to document and describe real-world treatment regimens and disease progression in multiple myeloma (MM) patients. Patients and Methods: Adult patients initiating any new MM therapy from October 2010 to October 2012 were eligible. A multistage patient/site recruitment model was applied to minimize the selection bias; enrollment was stratified by country, region, and practice type. The patient medical and disease features, treatment history, and remission status were recorded at baseline, and prospective data on treatment, efficacy, and safety were collected electronically every 3 months. Results: A total of 2358 patients were enrolled. Of these patients, 775 and 1583 did and did not undergo stem cell transplantation (SCT) at any time during treatment, respectively. Of the patients in the SCT and non-SCT groups, 49%, 21%, 14%, and 15% and 57%, 20%, 12% and 10% were enrolled at treatment line 1, 2, 3, and ≥ 4, respectively. In the SCT and non-SCT groups, 45% and 54% of the patients had received bortezomib-based therapy without thalidomide/lenalidomide, 12% and 18% had received thalidomide/lenalidomide-based therapy without bortezomib, and 30% and 4% had received bortezomib plus thalidomide/lenalidomide-based therapy as frontline treatment, respectively. The corresponding proportions of SCT and non-SCT patients in lines 2, 3, and ≥ 4 were 45% and 37%, 30% and 37%, and 12% and 3%, 33% and 27%, 35% and 32%, and 8% and 2%, and 27% and 27%, 27% and 23%, and 6% and 4%, respectively. In the SCT and non-SCT patients, the overall response rate was 86% to 97% and 64% to 85% in line 1, 74% to 78% and 59% to 68% in line 2, 55% to 83% and 48% to 60% in line 3, and 49% to 65% and 36% and 45% in line 4, respectively, for regimens that included bortezomib and/or thalidomide/lenalidomide. Conclusion: The results of our prospective study have revealed great diversity in the treatment regimens used to manage MM in real-life practice. This diversity was linked to factors such as novel agent accessibility and evolving treatment recommendations. Our results provide insight into associated clinical benefits.

AB - Multiple myeloma (MM) remains an incurable disease, with little information available on its management in real-world clinical practice. The results of the present prospective, noninterventional observational study revealed great diversity in the treatment regimens used to treat MM. Our results also provide data to inform health economic, pharmacoepidemiologic, and outcomes research, providing a framework for the design of protocols to improve the outcomes of patients with MM. Background: The present prospective, multinational, noninterventional study aimed to document and describe real-world treatment regimens and disease progression in multiple myeloma (MM) patients. Patients and Methods: Adult patients initiating any new MM therapy from October 2010 to October 2012 were eligible. A multistage patient/site recruitment model was applied to minimize the selection bias; enrollment was stratified by country, region, and practice type. The patient medical and disease features, treatment history, and remission status were recorded at baseline, and prospective data on treatment, efficacy, and safety were collected electronically every 3 months. Results: A total of 2358 patients were enrolled. Of these patients, 775 and 1583 did and did not undergo stem cell transplantation (SCT) at any time during treatment, respectively. Of the patients in the SCT and non-SCT groups, 49%, 21%, 14%, and 15% and 57%, 20%, 12% and 10% were enrolled at treatment line 1, 2, 3, and ≥ 4, respectively. In the SCT and non-SCT groups, 45% and 54% of the patients had received bortezomib-based therapy without thalidomide/lenalidomide, 12% and 18% had received thalidomide/lenalidomide-based therapy without bortezomib, and 30% and 4% had received bortezomib plus thalidomide/lenalidomide-based therapy as frontline treatment, respectively. The corresponding proportions of SCT and non-SCT patients in lines 2, 3, and ≥ 4 were 45% and 37%, 30% and 37%, and 12% and 3%, 33% and 27%, 35% and 32%, and 8% and 2%, and 27% and 27%, 27% and 23%, and 6% and 4%, respectively. In the SCT and non-SCT patients, the overall response rate was 86% to 97% and 64% to 85% in line 1, 74% to 78% and 59% to 68% in line 2, 55% to 83% and 48% to 60% in line 3, and 49% to 65% and 36% and 45% in line 4, respectively, for regimens that included bortezomib and/or thalidomide/lenalidomide. Conclusion: The results of our prospective study have revealed great diversity in the treatment regimens used to manage MM in real-life practice. This diversity was linked to factors such as novel agent accessibility and evolving treatment recommendations. Our results provide insight into associated clinical benefits.

KW - Bortezomib

KW - Global

KW - Observational study

KW - Routine practice

KW - Stem cell transplantation

UR - http://www.scopus.com/inward/record.url?scp=85050152150&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85050152150&partnerID=8YFLogxK

U2 - 10.1016/j.clml.2018.06.018

DO - 10.1016/j.clml.2018.06.018

M3 - Article

AN - SCOPUS:85050152150

JO - Clinical Lymphoma, Myeloma and Leukemia

JF - Clinical Lymphoma, Myeloma and Leukemia

SN - 2152-2669

ER -