■意义不明的单克隆丙种球蛋白病(MGUS)和闷烧的多发性骨髓瘤(SMM)是多发性骨髓瘤和相关疾病的无症状前兆。阴燃性多发性骨髓瘤与MGUS的区别在于采样时骨髓浆细胞(BMPC)的10%或更高,有更高的进展风险,需要专家管理。
■开发一种多变量预测模型,该模型可预测假定MGUS的人具有10%或更高的BMPC(根据骨髓标准,SMM或更差)的概率,以告知决定获得骨髓样本并将其性能与MayoClinic风险分层模型进行比较。
■iStopMM(冰岛屏幕,治疗或预防多发性骨髓瘤),一项基于人群的MGUS前瞻性筛查研究。(ClinicalTrials.gov:NCT03327597)。
■40岁或以上的冰岛人口。
■1043名IgG患者,IgA,轻链,和通过筛查和可解释的骨髓样本检测到的双克隆MGUS。
■未确定意义同种型的单克隆丙种球蛋白病;单克隆蛋白浓度;游离轻链比例;和总IgG,IgM,和IgA浓度用作预测因子。骨髓浆细胞分类为0%至4%,5%到9%,10%到14%,或15%或更高。
■SMM或更差的c统计量为0.85(95%CI,0.82至0.88),校准非常好(截距,-0.07;坡度,0.95)。在10%的阈值下,SMM或更坏的预测风险,灵敏度为86%,特异性为67%,阳性预测值为32%,阴性预测值为96%。与梅奥诊所模型相比,在合理的低风险阈值范围内,决定转诊进行抽样的净获益高出0.13~0.30.
■预测模型将需要外部验证。
■这种SMM或更差的准确预测模型是在假定为MGUS的人群中开发的,可用于推迟骨髓采样和转诊血液学。
■国际骨髓瘤基金会和欧洲研究理事会。
UNASSIGNED: 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.
UNASSIGNED: 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.
UNASSIGNED: iStopMM (Iceland Screens, Treats or Prevents Multiple Myeloma), a prospective population-based screening study of MGUS. (ClinicalTrials.gov: NCT03327597).
UNASSIGNED: Icelandic population of adults aged 40 years or older.
UNASSIGNED: 1043 persons with IgG, IgA, light-chain, and biclonal MGUS detected by screening and an interpretable bone marrow sample.
UNASSIGNED: 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.
UNASSIGNED: 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.
UNASSIGNED: The prediction model will require external validation.
UNASSIGNED: 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.
UNASSIGNED: International Myeloma Foundation and the European Research Council.