Connect MDS/AML: Design of the myelodysplastic syndromes and acute myeloid leukemia disease registry, a prospective observational cohort study

David P. Steensma, Mehrdad Abedi, Rafael Bejar, Christopher R. Cogle, Kathryn Foucar, Guillermo Garcia-Manero, Tracy I. George, David Grinblatt, Rami Komrokji, Xiaomei Ma, Jaroslaw Maciejewski, Daniel A. Pollyea, Michael R. Savona, Bart Scott, Mikkael A. Sekeres, Michael A. Thompson, Arlene S. Swern, Melissa Nifenecker, Mary M. Sugrue, Harry Erba

Research output: Contribution to journalArticle

7 Citations (Scopus)

Abstract

Background: Myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) are myeloid neoplasms in which outgrowth of neoplastic clones disrupts normal hematopoiesis. Some patients with unexplained persistent cytopenias may not meet minimal diagnostic criteria for MDS but an alternate diagnosis is not apparent; the term idiopathic cytopenia of undetermined significance (ICUS) has been used to describe this state. MDS and AML occur primarily in older patients who are often treated outside the clinical trial setting. Consequently, our understanding of the patterns of diagnostic evaluation, management, and outcomes of these patients is limited. Furthermore, there are few natural history studies of ICUS. To better understand how patients who have MDS, ICUS, or AML are managed in the routine clinical setting, the Connect MDS/AML Disease Registry, a multicenter, prospective, observational cohort study of patients newly diagnosed with these conditions has been initiated. Methods/Design: The Connect MDS/AML Disease Registry will capture diagnosis, risk assessment, treatment, and outcomes data for approximately 1500 newly diagnosed patients from approximately 150 community and academic sites in the United States in 4 cohorts: (1) lower-risk MDS (International Prognostic Scoring System [IPSS] low and intermediate-1 risk), with and without del(5q); (2) higher-risk MDS (IPSS intermediate-2 and high risk); (3) ICUS; and (4) AML in patients aged ≥ 55 years (excluding acute promyelocytic leukemia). Diagnosis will be confirmed by central review. Baseline patient characteristics, diagnostic patterns, treatment patterns, clinical outcomes, health economics outcomes, and patient-reported health-related quality of life will be entered into an electronic data capture system at enrollment and quarterly for 8 years. A tissue substudy to explore the relationship between karyotypes, molecular markers, and clinical outcomes will be conducted, and is optional for patients. Discussion: The Connect MDS/AML Disease Registry will be the first prospective, observational, non-interventional study in the United States to collect clinical information, patient-reported outcomes, and tissue samples from patients with MDS, ICUS, or AML receiving multiple therapies. Results from this registry may provide new insights into the relationship between diagnostic practices, treatment regimens, and outcomes in patients with these diseases and identify areas for future investigation.

Original languageEnglish (US)
Article number652
JournalBMC Cancer
Volume16
Issue number1
DOIs
StatePublished - Aug 19 2016

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Myelodysplastic Syndromes
Acute Myeloid Leukemia
Observational Studies
Registries
Cohort Studies
Acute Promyelocytic Leukemia
Hematopoiesis
Natural History
Karyotype
Information Systems
Clone Cells
Biomarkers
Economics
Quality of Life
Clinical Trials

Keywords

  • Acute myeloid leukemia
  • Biomarkers
  • Clinical outcomes
  • Clonal hematopoiesis of indeterminate potential (CHIP)
  • Idiopathic cytopenia of undetermined significance
  • Myelodysplastic syndromes
  • Patient-reported outcomes
  • Registry
  • Treatment patterns

ASJC Scopus subject areas

  • Genetics
  • Oncology
  • Cancer Research

Cite this

Connect MDS/AML : Design of the myelodysplastic syndromes and acute myeloid leukemia disease registry, a prospective observational cohort study. / Steensma, David P.; Abedi, Mehrdad; Bejar, Rafael; Cogle, Christopher R.; Foucar, Kathryn; Garcia-Manero, Guillermo; George, Tracy I.; Grinblatt, David; Komrokji, Rami; Ma, Xiaomei; Maciejewski, Jaroslaw; Pollyea, Daniel A.; Savona, Michael R.; Scott, Bart; Sekeres, Mikkael A.; Thompson, Michael A.; Swern, Arlene S.; Nifenecker, Melissa; Sugrue, Mary M.; Erba, Harry.

In: BMC Cancer, Vol. 16, No. 1, 652, 19.08.2016.

Research output: Contribution to journalArticle

Steensma, DP, Abedi, M, Bejar, R, Cogle, CR, Foucar, K, Garcia-Manero, G, George, TI, Grinblatt, D, Komrokji, R, Ma, X, Maciejewski, J, Pollyea, DA, Savona, MR, Scott, B, Sekeres, MA, Thompson, MA, Swern, AS, Nifenecker, M, Sugrue, MM & Erba, H 2016, 'Connect MDS/AML: Design of the myelodysplastic syndromes and acute myeloid leukemia disease registry, a prospective observational cohort study', BMC Cancer, vol. 16, no. 1, 652. https://doi.org/10.1186/s12885-016-2710-6
Steensma, David P. ; Abedi, Mehrdad ; Bejar, Rafael ; Cogle, Christopher R. ; Foucar, Kathryn ; Garcia-Manero, Guillermo ; George, Tracy I. ; Grinblatt, David ; Komrokji, Rami ; Ma, Xiaomei ; Maciejewski, Jaroslaw ; Pollyea, Daniel A. ; Savona, Michael R. ; Scott, Bart ; Sekeres, Mikkael A. ; Thompson, Michael A. ; Swern, Arlene S. ; Nifenecker, Melissa ; Sugrue, Mary M. ; Erba, Harry. / Connect MDS/AML : Design of the myelodysplastic syndromes and acute myeloid leukemia disease registry, a prospective observational cohort study. In: BMC Cancer. 2016 ; Vol. 16, No. 1.
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abstract = "Background: Myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) are myeloid neoplasms in which outgrowth of neoplastic clones disrupts normal hematopoiesis. Some patients with unexplained persistent cytopenias may not meet minimal diagnostic criteria for MDS but an alternate diagnosis is not apparent; the term idiopathic cytopenia of undetermined significance (ICUS) has been used to describe this state. MDS and AML occur primarily in older patients who are often treated outside the clinical trial setting. Consequently, our understanding of the patterns of diagnostic evaluation, management, and outcomes of these patients is limited. Furthermore, there are few natural history studies of ICUS. To better understand how patients who have MDS, ICUS, or AML are managed in the routine clinical setting, the Connect MDS/AML Disease Registry, a multicenter, prospective, observational cohort study of patients newly diagnosed with these conditions has been initiated. Methods/Design: The Connect MDS/AML Disease Registry will capture diagnosis, risk assessment, treatment, and outcomes data for approximately 1500 newly diagnosed patients from approximately 150 community and academic sites in the United States in 4 cohorts: (1) lower-risk MDS (International Prognostic Scoring System [IPSS] low and intermediate-1 risk), with and without del(5q); (2) higher-risk MDS (IPSS intermediate-2 and high risk); (3) ICUS; and (4) AML in patients aged ≥ 55 years (excluding acute promyelocytic leukemia). Diagnosis will be confirmed by central review. Baseline patient characteristics, diagnostic patterns, treatment patterns, clinical outcomes, health economics outcomes, and patient-reported health-related quality of life will be entered into an electronic data capture system at enrollment and quarterly for 8 years. A tissue substudy to explore the relationship between karyotypes, molecular markers, and clinical outcomes will be conducted, and is optional for patients. Discussion: The Connect MDS/AML Disease Registry will be the first prospective, observational, non-interventional study in the United States to collect clinical information, patient-reported outcomes, and tissue samples from patients with MDS, ICUS, or AML receiving multiple therapies. Results from this registry may provide new insights into the relationship between diagnostic practices, treatment regimens, and outcomes in patients with these diseases and identify areas for future investigation.",
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T2 - Design of the myelodysplastic syndromes and acute myeloid leukemia disease registry, a prospective observational cohort study

AU - Steensma, David P.

AU - Abedi, Mehrdad

AU - Bejar, Rafael

AU - Cogle, Christopher R.

AU - Foucar, Kathryn

AU - Garcia-Manero, Guillermo

AU - George, Tracy I.

AU - Grinblatt, David

AU - Komrokji, Rami

AU - Ma, Xiaomei

AU - Maciejewski, Jaroslaw

AU - Pollyea, Daniel A.

AU - Savona, Michael R.

AU - Scott, Bart

AU - Sekeres, Mikkael A.

AU - Thompson, Michael A.

AU - Swern, Arlene S.

AU - Nifenecker, Melissa

AU - Sugrue, Mary M.

AU - Erba, Harry

PY - 2016/8/19

Y1 - 2016/8/19

N2 - Background: Myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) are myeloid neoplasms in which outgrowth of neoplastic clones disrupts normal hematopoiesis. Some patients with unexplained persistent cytopenias may not meet minimal diagnostic criteria for MDS but an alternate diagnosis is not apparent; the term idiopathic cytopenia of undetermined significance (ICUS) has been used to describe this state. MDS and AML occur primarily in older patients who are often treated outside the clinical trial setting. Consequently, our understanding of the patterns of diagnostic evaluation, management, and outcomes of these patients is limited. Furthermore, there are few natural history studies of ICUS. To better understand how patients who have MDS, ICUS, or AML are managed in the routine clinical setting, the Connect MDS/AML Disease Registry, a multicenter, prospective, observational cohort study of patients newly diagnosed with these conditions has been initiated. Methods/Design: The Connect MDS/AML Disease Registry will capture diagnosis, risk assessment, treatment, and outcomes data for approximately 1500 newly diagnosed patients from approximately 150 community and academic sites in the United States in 4 cohorts: (1) lower-risk MDS (International Prognostic Scoring System [IPSS] low and intermediate-1 risk), with and without del(5q); (2) higher-risk MDS (IPSS intermediate-2 and high risk); (3) ICUS; and (4) AML in patients aged ≥ 55 years (excluding acute promyelocytic leukemia). Diagnosis will be confirmed by central review. Baseline patient characteristics, diagnostic patterns, treatment patterns, clinical outcomes, health economics outcomes, and patient-reported health-related quality of life will be entered into an electronic data capture system at enrollment and quarterly for 8 years. A tissue substudy to explore the relationship between karyotypes, molecular markers, and clinical outcomes will be conducted, and is optional for patients. Discussion: The Connect MDS/AML Disease Registry will be the first prospective, observational, non-interventional study in the United States to collect clinical information, patient-reported outcomes, and tissue samples from patients with MDS, ICUS, or AML receiving multiple therapies. Results from this registry may provide new insights into the relationship between diagnostic practices, treatment regimens, and outcomes in patients with these diseases and identify areas for future investigation.

AB - Background: Myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) are myeloid neoplasms in which outgrowth of neoplastic clones disrupts normal hematopoiesis. Some patients with unexplained persistent cytopenias may not meet minimal diagnostic criteria for MDS but an alternate diagnosis is not apparent; the term idiopathic cytopenia of undetermined significance (ICUS) has been used to describe this state. MDS and AML occur primarily in older patients who are often treated outside the clinical trial setting. Consequently, our understanding of the patterns of diagnostic evaluation, management, and outcomes of these patients is limited. Furthermore, there are few natural history studies of ICUS. To better understand how patients who have MDS, ICUS, or AML are managed in the routine clinical setting, the Connect MDS/AML Disease Registry, a multicenter, prospective, observational cohort study of patients newly diagnosed with these conditions has been initiated. Methods/Design: The Connect MDS/AML Disease Registry will capture diagnosis, risk assessment, treatment, and outcomes data for approximately 1500 newly diagnosed patients from approximately 150 community and academic sites in the United States in 4 cohorts: (1) lower-risk MDS (International Prognostic Scoring System [IPSS] low and intermediate-1 risk), with and without del(5q); (2) higher-risk MDS (IPSS intermediate-2 and high risk); (3) ICUS; and (4) AML in patients aged ≥ 55 years (excluding acute promyelocytic leukemia). Diagnosis will be confirmed by central review. Baseline patient characteristics, diagnostic patterns, treatment patterns, clinical outcomes, health economics outcomes, and patient-reported health-related quality of life will be entered into an electronic data capture system at enrollment and quarterly for 8 years. A tissue substudy to explore the relationship between karyotypes, molecular markers, and clinical outcomes will be conducted, and is optional for patients. Discussion: The Connect MDS/AML Disease Registry will be the first prospective, observational, non-interventional study in the United States to collect clinical information, patient-reported outcomes, and tissue samples from patients with MDS, ICUS, or AML receiving multiple therapies. Results from this registry may provide new insights into the relationship between diagnostic practices, treatment regimens, and outcomes in patients with these diseases and identify areas for future investigation.

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KW - Biomarkers

KW - Clinical outcomes

KW - Clonal hematopoiesis of indeterminate potential (CHIP)

KW - Idiopathic cytopenia of undetermined significance

KW - Myelodysplastic syndromes

KW - Patient-reported outcomes

KW - Registry

KW - Treatment patterns

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