Development of a Multivariable Model to Predict the Need for Bone Marrow Sampling in Persons With Monoclonal Gammopathy of Undetermined Significance : A Cohort Study Nested in a Clinical Trial.

Autor: Eythorsson E; Landspítali-The National University Hospital of Iceland and Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.E., S.R., P.T.O., B.A.A., R.P., O.S.I., S.Y.K.)., Rognvaldsson S; Landspítali-The National University Hospital of Iceland and Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.E., S.R., P.T.O., B.A.A., R.P., O.S.I., S.Y.K.)., Thorsteinsdottir S; Faculty of Medicine, University of Iceland, Reykjavík, Iceland, and Department of Hematology, Rigshospitalet, Copenhagen, Denmark (S.T.)., Einarsson Long T; Faculty of Medicine, University of Iceland, Reykjavík, Iceland, and Skåne University Hospital, Lund, Sweden (T.E.L.)., Reed ER; Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.R.R., G.A.S., A.R.T., G.K.G., A.O., J.S., T.J.L.)., Sigurdardottir GA; Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.R.R., G.A.S., A.R.T., G.K.G., A.O., J.S., T.J.L.)., Vidarsson B; Landspítali-The National University Hospital of Iceland, Reykjavík, Iceland (B.V., M.S., I.O., I.T., S.V.S., F.S., H.S.)., Onundarson PT; Landspítali-The National University Hospital of Iceland and Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.E., S.R., P.T.O., B.A.A., R.P., O.S.I., S.Y.K.)., Agnarsson BA; Landspítali-The National University Hospital of Iceland and Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.E., S.R., P.T.O., B.A.A., R.P., O.S.I., S.Y.K.)., Sigurdardottir M; Landspítali-The National University Hospital of Iceland, Reykjavík, Iceland (B.V., M.S., I.O., I.T., S.V.S., F.S., H.S.)., Olafsson I; Landspítali-The National University Hospital of Iceland, Reykjavík, Iceland (B.V., M.S., I.O., I.T., S.V.S., F.S., H.S.)., Thorsteinsdottir I; Landspítali-The National University Hospital of Iceland, Reykjavík, Iceland (B.V., M.S., I.O., I.T., S.V.S., F.S., H.S.)., Sveinsdottir SV; Landspítali-The National University Hospital of Iceland, Reykjavík, Iceland (B.V., M.S., I.O., I.T., S.V.S., F.S., H.S.)., Sigurdsson F; Landspítali-The National University Hospital of Iceland, Reykjavík, Iceland (B.V., M.S., I.O., I.T., S.V.S., F.S., H.S.)., Thordardottir AR; Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.R.R., G.A.S., A.R.T., G.K.G., A.O., J.S., T.J.L.)., Palsson R; Landspítali-The National University Hospital of Iceland and Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.E., S.R., P.T.O., B.A.A., R.P., O.S.I., S.Y.K.)., Indridason OS; Landspítali-The National University Hospital of Iceland and Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.E., S.R., P.T.O., B.A.A., R.P., O.S.I., S.Y.K.)., Jonsson A; Akureyri Hospital, Akureyri, Iceland (A.J.)., Gislason GK; Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.R.R., G.A.S., A.R.T., G.K.G., A.O., J.S., T.J.L.)., Olafsson A; Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.R.R., G.A.S., A.R.T., G.K.G., A.O., J.S., T.J.L.)., Sigurdsson J; Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.R.R., G.A.S., A.R.T., G.K.G., A.O., J.S., T.J.L.)., Steingrimsdottir H; Landspítali-The National University Hospital of Iceland, Reykjavík, Iceland (B.V., M.S., I.O., I.T., S.V.S., F.S., H.S.)., Hultcrantz M; Myeloma Service, Memorial Sloan Kettering Cancer Center, New York, New York (M.H.)., Durie BGM; Cedars-Sinai Samuel Oschin Cancer Center, Los Angeles, California (B.G.M.D.)., Harding S; The Binding Site, Birmingham, West Midlands, United Kingdom (S.H.)., Landgren O; Sylvester Comprehensive Cancer Center, University of Miami, Miami, Florida (O.L.)., Aspelund T; Center for Public Health Sciences, University of Iceland, Reykjavík, Iceland (T.A.)., Love TJ; Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.R.R., G.A.S., A.R.T., G.K.G., A.O., J.S., T.J.L.)., Kristinsson SY; Landspítali-The National University Hospital of Iceland and Faculty of Medicine, University of Iceland, Reykjavík, Iceland (E.E., S.R., P.T.O., B.A.A., R.P., O.S.I., S.Y.K.).
Jazyk: angličtina
Zdroj: Annals of internal medicine [Ann Intern Med] 2024 Apr; Vol. 177 (4), pp. 449-457. Date of Electronic Publication: 2024 Apr 02.
DOI: 10.7326/M23-2540
Abstrakt: Background: Monoclonal gammopathy of undetermined significance (MGUS) and smoldering multiple myeloma (SMM) are asymptomatic precursor conditions to multiple myeloma and related disorders. Smoldering multiple myeloma is distinguished from MGUS by 10% or greater bone marrow plasma cells (BMPC) on sampling, has a higher risk for progression, and requires specialist management.
Objective: To develop a multivariable prediction model that predicts the probability that a person with presumed MGUS has 10% or greater BMPC (SMM or worse by bone marrow criteria) to inform the decision to obtain a bone marrow sample and compare its performance to the Mayo Clinic risk stratification model.
Design: iStopMM (Iceland Screens, Treats or Prevents Multiple Myeloma), a prospective population-based screening study of MGUS. (ClinicalTrials.gov: NCT03327597).
Setting: Icelandic population of adults aged 40 years or older.
Patients: 1043 persons with IgG, IgA, light-chain, and biclonal MGUS detected by screening and an interpretable bone marrow sample.
Measurements: Monoclonal gammopathy of undetermined significance isotype; monoclonal protein concentration; free light-chain ratio; and total IgG, IgM, and IgA concentrations were used as predictors. Bone marrow plasma cells were categorized as 0% to 4%, 5% to 9%, 10% to 14%, or 15% or greater.
Results: The c-statistic for SMM or worse was 0.85 (95% CI, 0.82 to 0.88), and calibration was excellent (intercept, -0.07; slope, 0.95). At a threshold of 10% predicted risk for SMM or worse, sensitivity was 86%, specificity was 67%, positive predictive value was 32%, and negative predictive value was 96%. Compared with the Mayo Clinic model, the net benefit for the decision to refer for sampling was between 0.13 and 0.30 higher over a range of plausible low-risk thresholds.
Limitation: The prediction model will require external validation.
Conclusion: This accurate prediction model for SMM or worse was developed in a population-based cohort of persons with presumed MGUS and may be used to defer bone marrow sampling and referral to hematology.
Primary Funding Source: International Myeloma Foundation and the European Research Council.
Competing Interests: Disclosures: Disclosures can be viewed at www.acponline.org/authors/icmje/ConflictOfInterestForms.do?msNum=M23-2540.
Databáze: MEDLINE