scholarly journals Treatment Outcome for Symptomatic Patients with Clonal Cytopenia of Undetermined Significance: A Single-Institution Retrospective Study

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 44-44
Author(s):  
Zhuoer Xie ◽  
Ahmad Nanaa ◽  
Antoine Saliba ◽  
Rong He ◽  
Phuong L. Nguyen ◽  
...  
Keyword(s):  
Native American ◽  
Blood Cell ◽  
Vitamin B12 ◽  
Platelet Transfusion ◽  
Single Institution ◽  
Treatment Type ◽  
Transfusion Dependency ◽  
Undetermined Significance ◽  
Dependency Rate

Background Clonal cytopenia of undetermined significance (CCUS) is a newly described entity with a high probability to progress into myeloid disorders upon follow-up (Malcovati et al, Blood 2017). Little data is known on how to effectively treat patients with CCUS. We hereby describe a single institution experience in managing symptomatic CCUS patients. Methods Patients who had CCUS and underwent active treatment were identified after receiving IRB approval. CCUS diagnosis was rendered if patients had non-diagnostic bone marrow biopsy evaluation combined with evidence of pathogenic myeloid somatic mutation using our institution next generation sequencing (NGS) panel (OncoHeme, Mayo Clinic). "Symptomatic patients" is determined by patients who need therapy beyond blood transfusion. Treatment type is based on physicians' choice. Clinical and lab results were collected. Response and progression were graded based on the MDS IWG 2006 criteria. The date of treatment initiation was used for overall survival (OS) and progression (either to myeloid neoplasm or worsening cytopenias) free survival (PFS) calculation. Stata 14.1(StatCorp) was used for data analysis. Results 1) Cohort characteristics: Between 2015 and 2020, 24 patients met inclusion criteria with median age of 72 years (range: 24-87). Twenty (83%) were men. 15 (63%) were white, 1 (4%) African American, 1 (4%) Native American and 7 unknown (33%). Eighty percent had ECOG PS≤1. Four (17%) patients had prior other hematology disorders and 3 (13%) had solid tumor. Two (8%) patients had previous radiation therapy, 2 (8%) received chemotherapy and 1 (4%) received allogeneic stem cell transplantation. At the time of treatment, the median of hemoglobin was 8.9 (range 6.8-13.7) g/dL, white blood cell 2.9 (range 1.4-12.3) *109/L, and platelet 76 (range 8-407) *109/L. Red blood cell (RBC) and platelet transfusion dependency rate were 54% and 29%, respectively. We identified 23 different mutations. Most common mutations occurred in SRSF2 (N=5, 21%), TET2 (N=5, 21%), U2AF1 (N=5, 21%), and TP53 (N=4, 17%). Ten (42%) patients had 1 mutation, and 14 (58%) had ≥2 mutations. Treatment included hypomethylating agents (HMA) (N=10, 42%), growth factors (N=11, 46%), steroid (N=4, 17%), testosterone (N=2, 8%), cyclosporine (N=2, 8%), rituximab (N=1, 4%), immunoglobulin (N=1, 4%), vitamin B12 and iron (N=1, 4%). Seven (29%) patients received ≥2 treatments. Median follow up duration was 17.5 months (range 8.9-26.1). Median time from the diagnosis to treatment was 2.1 months (range 0-26.8). 2) Molecular correlation with response The overall response rate (RR) was 50% (hematological or symptomatic improvement). Most responders (67%) were treated with HMA (p=0.013). HMA treatment effect was not significantly associated with DNA methylation genes (DNMT3A, IDH1, IDH2 and TET2, all p>0.05). The RR was 100% for the three patients with IDH1 mutation (treated with HMA, steroid and erythropoietin stimulating agent (ESA), respectively). The RR was 100% for the 3 patients with ZRSR2 mutation (2 HMA, 1 testosterone). The RR was 0% for 3 patients with SF3B1 mutation (1 testosterone and ESA, 1 vitamin B12 and iron, and 1 HMA). The number of mutations had no impact on RR (p=0.41). Table demonstrates the RR for each treatment. 3) Survival and progression outcome At the last day of follow up, RBC and platelet transfusion dependency rate were 38% and 25%, respectively. The median PFS was 16.9 months (95% CI: 7.5-65.3). Six patients (25%) progressed to myeloid malignancy with 4 myelodysplastic syndrome (MDS) and 2 acute myeloid leukemia (AML). Five patients progressed with worsening cytopenias. Among the 4 MDS patients, they had SF3B1, TET2, and ASXL1 mutations at the diagnosis of CCUS. Within the 2 AML pts, 1 had RUNX1 mutation, 1 had BCOR, DNMT3A, PHF6, RUNX1, SF3B1, STAG2, and TET2 mutations at the diagnosis of CCUS. The median OS was not reached with an estimated 2 year OS 77%. Three (13%) patients progressed with cytopenia and died. Their NGS at CCUS diagnosis showed ASXL1, TP53 and U2AF1 mutations, respectively. Conclusion Patients with CCUS responded to available treatments used for myeloid diseases. HMA were effective in this cohort. Mutation profile may have an impact on treatment response. Studies of larger cohorts are needed to validate our findings. Additionally, development of response and progression criteria would be helpful for such patients for reporting treatment outcomes. Disclosures Shah: Dren Bio: Consultancy.

scholarly journals IDH2 Inhibitor Therapy in Relapsed and Refractory Acute Myeloid Leukemia: A Single Institution Experience

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 43-44
Author(s):  
Marissa Li ◽  
Erika Morsia ◽  
Christopher E. Wee ◽  
Kristen McCullough ◽  
Aref Al-Kali ◽  
...  
Keyword(s):  
Myeloid Leukemia ◽  
Platelet Transfusion ◽  
Median Overall Survival ◽  
Response Assessment ◽  
Complete Response ◽  
Transfusion Independence ◽  
Transfusion Dependency ◽  
Initiation Of Therapy ◽  
Acute Myeloid

Introduction: Mutations in the gene isocitrate dehydrogenase 2 (IDH2) occur in ~12-15% of patients with acute myeloid leukemia (AML). Enasidenib, an oral small molecule selective targeted inhibitor of IDH2 enzymes, was approved forIDH2mutated relapsed/refractory AML (RR-AML) in 2017 (Stein et. al., 2017). There is limited data regarding its efficacy and toxicity outside the context of clinical trials. Using Mayo Clinic's cohort of AML patients, we provide a real-world look at outcomes forIDH2mutated RR-AML patients treated with enasidenib. Methods: A total of 13 patients treated with enasidenib for RR-AML were identified, all of which experienced at least one relapse following induction chemotherapy. Response assessment was based on European Leukemia Net (ELN) 2017 response criteria.Complete blood counts including blast percentage and transfusion dependency were obtained at AML diagnosis, initiation of enasidenib, 2 months and 6 months following initiation of therapy. Transfusion dependency was defined by the need for either a red cell or platelet transfusion within 4 weeks of the designated time frame. We compared the clinical characteristics of patients who achieved complete response (CR) (n=4) to those not achieving CR (n=9), using the Mann-Whitney U test for numerical variables and the Fischer's Exact test for quantitative data. Results: The median age at diagnosis of our 13 patients was 68 years (range 60-82), with a male predilection (n=11) (Table 1). Cytogenetic and molecular genetic findings are provided in Table 2. Complete response (CR) was achieved in 4 (30.7%) patients by cycle 2 and 2 of those patients (50%) proceeded to an allogeneic hematopoietic stem cell transplant (AHSCT). The median time from diagnosis to initiation of treatment for patients in CR was 3.5 months (range 1-7), and for patients not achieving CR was 13 months (range 6-53; p=0.048), with median time of follow up after treatment initiation of 23.5 months (range, 5-36) and 7 months (range, 1-14), respectively. Among 8 patients who were transfusion dependent at baseline, all the CR patients (2/2) achieved red blood cell and platelet transfusion independence 6 months after treatment, instead only (3/6) 50% of those not in CR achieved transfusion independence (p=0.028). An initial trajectory of improvement in counts is noted from the time of diagnosis to the 2 month mark post treatment with 75% of the responder cohort showing improvement in counts which was durable through the 6 month mark, whereas 55% of those that did not respond had worsening counts at month 2 and died prior to 6 month follow up. At the time of data cut-off, the median duration of CR was 14 months (range, 2-33), with 3 of 4 (75%) of responders maintaining CR. Figure 1 details overall responses and outcomes in all 13 patients. None of the patients in our study experienced differentiation syndrome. Five patients were hospitalized in the course of treatment for infectious complications. Two patients were noted to have hyperbilirubinemia however there was no clear indication that this was secondary to enasidenib Median overall survival for all 13 patients was 21 months (range, 6-53 months) with leukemia free survival of 7 months (range, 5-19 months). Furthermore, median overall survival for patients in CR was 27 months (range, 6-42), and those not in CR was 16 months (range, 6-53) (p=0.20). In addition, we observed that 75% of patients who attained CR are alive compared to 11.1% of those not in CR (p = 0.052). Conclusion: We demonstrate a superior CR rate of 30.7% with enasidenib amongstIDH2mutated RR-AML patients compared with clinical trial data showing CR rate of 19.6% (Stein et. al., 2019) despite our patients being heavily pre-treated with median number of two prior therapies (range; 1-5) including prior AHSCT in 2 patients. Moreover, a subset of patients (2 of 4 in CR) were able to undergo ASCHT. Finally, the achievement of CR at 2 months after initiation of therapy serves as an appropriate time point for response assessment. Disclosures No relevant conflicts of interest to declare.

Mean Corpuscular Volume as An Early Predictor of Malignant Evolution of Monoclonal Gammopathy of Undetermined Significance

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 1925-1925
Author(s):  
Mustapha A. Khalife ◽  
Philip Kuriakose ◽  
Marwa Hammoud
Keyword(s):  
Monoclonal Gammopathy ◽  
Mean Corpuscular Volume ◽  
Red Cell ◽  
Single Institution ◽  
Chi Square ◽  
Chi Square Test ◽  
Group 2 ◽  
Undetermined Significance ◽  
Group 1

Abstract Abstract 1925 Introduction: Monoclonal gammopathy of undetermined significance (MGUS) is an asymptomatic premalignant clonal plasma cell proliferative disorder. It occurs in over 3 percent of the general population over the age of 50. MGUS can progress to multiple myeloma (MM) or related cancers at a rate of about 1.0 to 1.5 percent per year. There are no findings at diagnosis of MGUS that reliably distinguish patients who will remain stable from those who will progress to a malignant disease. Size and type of serum monoclonal protein, as well as serum free light chain ratio have been suggested in some studies as independent risk factors for disease progression. In this study, we tried to analyze the relationship between Mean Corpuscular Volume (MCV) and the risk of progression of MGUS into a malignant lymphoproliferative condition. Methods: A total of 1744 patients, from a single institution, with a first diagnosis of MGUS from April 18th, 1995 till June 24th, 2010 were included in this study. Patients analyzed were divided into two groups. Group 1 consisted of 345 patients with an elevated MCV (>100fL/red cell) and group 2 consisted of 1399 patients with a normal MCV (between 80fL/red cell and 100fL/red cell). Patients with short follow up (<3 months), low B12/folate levels, hypothyroidism, liver disease or a serum monoclonal protein >1.5g/dL were excluded. The Chi-square test was used to compare the proportion of patients who progressed in group 1 relative to group 2. Results: After a median follow up of 51 and 58 months for the high MCV and normal MCV respectively, there were 78 progressions (22.6%) versus 267 non-progressions (77.4%) among the high MCV patients (Group1) and 110 progressions (7.9%) versus 1289 non-progressions (92.1%) among the normal MCV patients (Group 2). The difference in progression between the two groups was statistically significant (Chi-square test p-value < 0.001). The odds ratio for the progression comparison of the high versus normal MCV groups was 3.4 with a 95% confidence interval of 2.5 to 4.7. Conclusion: In our single institution, retrospective study, there seems to be a higher incidence of malignant transformation in the subset of patients with MGUS having an elevated MCV. MCV is a cost effective method that might help identify, at presentation, patients with benign monoclonal gammopathies requiring stricter monitoring. Disclosures: No relevant conflicts of interest to declare.

Long-term outcomes of lumbar microdiscectomy in the pediatric population: a large single-institution case series

2019 ◽  
Vol 24 (5) ◽  
pp. 549-557
Author(s):  
Malia McAvoy ◽  
Heather J. McCrea ◽  
Vamsidhar Chavakula ◽  
Hoon Choi ◽  
Wenya Linda Bi ◽  
...  
Keyword(s):  
Conservative Management ◽  
Functional Outcomes ◽  
Pediatric Patients ◽  
Clinical Presentation ◽  
Case Series ◽  
Csf Leak ◽  
Single Institution ◽  
The Mean

OBJECTIVEFew studies describe long-term functional outcomes of pediatric patients who have undergone lumbar microdiscectomy (LMD) because of the rarity of pediatric disc herniation and the short follow-up periods. The authors analyzed risk factors, clinical presentation, complications, and functional outcomes of a single-institution series of LMD patients over a 19-year period.METHODSA retrospective case series was conducted of pediatric LMD patients at a large pediatric academic hospital from 1998 to 2017. The authors examined premorbid risk factors, clinical presentation, physical examination findings, type and duration of conservative management, indications for surgical intervention, complications, and postoperative outcomes.RESULTSOver the 19-year study period, 199 patients underwent LMD at the authors’ institution. The mean age at presentation was 16.0 years (range 12–18 years), and 55.8% were female. Of these patients, 70.9% participated in competitive sports, and among those who did not play sports, 65.0% had a body mass index greater than 25 kg/m2. Prior to surgery, conservative management had failed in 98.0% of the patients. Only 3 patients (1.5%) presented with cauda equina syndrome requiring emergent microdiscectomy. Complications included 4 cases of postoperative CSF leak (2.0%), 1 case of a noted intraoperative CSF leak, and 3 cases of wound infection (1.5%). At the first postoperative follow-up appointment, minimal or no pain was reported by 93.3% of patients. The mean time to return to sports was 9.8 weeks. During a mean follow-up duration of 8.2 years, 72.9% of patients did not present again after routine postoperative appointments. The total risk of reoperation was a rate of 7.5% (3.5% of patients underwent reoperation for the same level; 4.5% underwent adjacent-level decompression, and one patient [0.5%] ultimately underwent a fusion).CONCLUSIONSMicrodiscectomy is a safe and effective treatment for long-term relief of pain and return to daily activities among pediatric patients with symptomatic lumbar disc disease in whom conservative management has failed.

Red Blood Cell Indices in Relation to Post-stroke Psychiatric Disorders: A Longitudinal Study in a Follow-up Stroke Clinic

2020 ◽  
Vol 17 (3) ◽  
pp. 218-223
Author(s):  
Haichao Wang ◽  
Li Gong ◽  
Xiaomei Xia ◽  
Qiong Dong ◽  
Aiping Jin ◽  
...  
Keyword(s):  
Regression Analysis ◽  
Ischemic Stroke ◽  
Blood Cell ◽  
Cox Regression ◽  
Depression And Anxiety ◽  
Cox Regression Analysis ◽  
Post Stroke ◽  
Rbc Indices ◽  
Post Stroke Depression

Background: Depression and anxiety after stroke are common conditions that are likely to be neglected. Abnormal red blood cell (RBC) indices may be associated with neuropsychiatric disorders. However, the association of RBC indices with post-stroke depression (PSD) and poststroke anxiety (PSA) has not been sufficiently investigated. Methods: We aimed to investigate the trajectory of post-stroke depression and anxiety in our follow- up stroke clinic at 1, 3, and 6 months, and the association of RBC indices with these. One hundred and sixty-two patients with a new diagnosis of ischemic stroke were followed up at 1, 3, and 6 months, and underwent Patient Health Questionnaire-9 (PHQ-9) and the general anxiety disorder 7-item (GAD-7) questionnaire for evaluation of depression and anxiety, respectively. First, we used Kaplan-Meier analysis to investigate the accumulated incidences of post-stroke depression and post-stroke anxiety. Next, to explore the association of RBC indices with psychiatric disorders after an ischemic stroke attack, we adjusted for demographic and vascular risk factors using multivariate Cox regression analysis. Results: Of the 162 patients with new-onset of ischemic stroke, we found the accumulated incidence rates of PSD (1.2%, 17.9%, and 35.8%) and PSA (1.2%, 13.6%, and 15.4%) at 1, 3, and 6 months, respectively. The incident PSD and PSA increased 3 months after a stroke attack. Multivariate Cox regression analysis indicated independent positive associations between PSD risk and higher mean corpuscular volume (MCV) (OR=1.42, 95% CI=1.16-1.76), older age (OR=2.63, 95% CI=1.16-5.93), and a negative relationship between male sex (OR=0.95, 95% CI=0.91-0.99) and PSA. Conclusion: The risks of PSD and PSA increased substantially 3 months beyond stroke onset. Of the RBC indices, higher MCV, showed an independent positive association with PSD.

scholarly journals Vitamin Intake and Loss of Muscle Mass in Older People with Type 2 Diabetes: A Prospective Study of the KAMOGAWA-DM Cohort

Nutrients ◽  
2021 ◽  
Vol 13 (7) ◽  
pp. 2335
Author(s):  
Fuyuko Takahashi ◽  
Yoshitaka Hashimoto ◽  
Ayumi Kaji ◽  
Ryosuke Sakai ◽  
Yuka Kawate ◽  
...  
Keyword(s):  
Type 2 Diabetes ◽  
Vitamin D ◽  
Older People ◽  
Vitamin B12 ◽  
Muscle Mass ◽  
Vitamin B1 ◽  
Vitamin D Intake ◽  
Glucagon Like Peptide 1

The aim of this prospective cohort study was to examine the relationships between the intakes of various vitamins and the loss of muscle mass in older people with type 2 diabetes (T2DM). The change in skeletal muscle mass index (SMI, kg/m2) (kg/m2/year) was defined as follows: (SMI at baseline (kg/m2) − SMI at follow-up (kg/m2))/follow-up period (year). The rate of SMI reduction (%) was calculated as follows (the change in SMI (kg/m2/year)/SMI at baseline (kg/m2)) × 100. The rate of SMI reduction ≥ 1.2% was considered as the loss of muscle mass. Among 197 people with T2DM, 47.2% of them experienced the loss of muscle mass at the 13.7 ± 5.2 month follow-up. Vitamin B1 (0.8 ± 0.3 vs. 0.8 ± 0.3 mg/day, p = 0.031), vitamin B12 (11.2 ± 8.3 vs. 13.4 ± 7.5 μg/day, p = 0.049), and vitamin D (16.5 ± 12.2 vs. 21.6 ± 13.0 μg/day, p = 0.004) intakes in people with the loss of muscle mass were significantly lower than those without. Vitamin D intake was related to the loss of muscle mass after adjusting for sex, age, exercise, alcohol, smoking, body mass index, SMI, glucagon-like peptide-1 agonist, sodium glucose cotransporter-2 inhibitor, insulin, HbA1c, creatinine, energy intake, and protein intake (adjusted odds ratio 0.93, 95% confidence interval: 0.88–0.97, p = 0.003). This study showed that vitamin D intake was related to the loss of muscle mass in older people with T2DM. Vitamin B12 intake tended to be related to the loss of muscle mass, although vitamin A, vitamin B2, vitamin B6, vitamin C, and vitamin E intake were not related.

scholarly journals Behind the clock: elucidating factors contributing to longer clinic appointment duration and patient wait time

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Daniel Jonathan Kagedan ◽  
Stephen B. Edge ◽  
Kazuaki Takabe
Keyword(s):  
Wait Time ◽  
High Volume ◽  
Advanced Practice ◽  
Single Institution ◽  
Clinic Appointment ◽  
Nurse Assessment ◽  
Patient Wait Time ◽  
Encounter Time ◽  
Examination Room

Abstract Background Longer wait time in ambulatory clinics can disrupt schedules and decrease satisfaction. We investigated factors associated with patient wait time (WT, check-in to examination room placement), approximate clinician time (ACT, completion of nurse assessment to check-out), and total appointment length (TAL, check-in to check-out). Methods A single-institution retrospective study was conducted of breast surgery clinic patients, 2017–2019, using actual encounter times. A before/after analysis compared a five-day 8 hour/day (from a four-day 10 hour/day) advanced practice provider (APP) work-week. Non-parametric tests were used, and medians with interquartile ranges (IQRs) reported. Results 15,265 encounters were identified. Overall WT was 15.0 minutes (IQR:6.0–32.0), ACT 49.0 minutes (IQR:31.0–79.0) and TAL 84.0 minutes (IQR:57.0-124.0). Trainees were associated with 30.0 minutes longer ACT (p < 0.0001); this increased time was greatest for follow-up appointments, least for new patients. Patients arriving > 5 minutes late (versus on-time) experienced shorter WT (11.0 vs. 15.0 minutes, p < 0.0001) and ACT (43.0 vs. 53.0 minutes, p < 0.0001). Busier days (higher encounter volume:APP ratios) demonstrated increased encounter times. After transitioning to a five-day APP work-week, ACT decreased. Conclusions High-volume clinics and trainee involvement prolong ambulatory encounters. Increasing APP assistance, altering work schedules, and assigning follow-up appointments to non-trainees may decrease encounter time.

Ultrasound Biomicroscopy Documented Anterior Uveal Melanoma Regression after Ruthenium-106 Plaque Therapy

2021 ◽  
pp. 1-9
Author(s):  
Biljana Kuzmanović Elabjer ◽  
Mladen Bušić ◽  
Andrej Pleše ◽  
Mirjana Bjeloš ◽  
Daliborka Miletić ◽  
...  
Keyword(s):  
Uveal Melanoma ◽  
Tumor Regression ◽  
Corneal Thickness ◽  
Case Series ◽  
Ultrasound Biomicroscopy ◽  
Close Monitoring ◽  
Single Institution ◽  
Retrospective Case ◽  
Caucasian Patients

<b><i>Introduction:</i></b> Ultrasound biomicroscopy (UBM) is the only widely used method for the evaluation of anterior uveal melanoma (AUM). <b><i>Objective:</i></b> Documentation of regression of AUM treated with ruthenium-106 (Ru-106) plaque types CCB and CCC using UBM. <b><i>Methods:</i></b> This single institution-based retrospective case series involved 10 Caucasian patients with AUM followed after brachytherapy with UBM from January 2014 until February 2019. The largest prominence of the tumor perpendicular to the sclera or the cornea (including scleral/corneal thickness) (<i>D</i>) and the largest basal dimension (<i>B</i>) were measured in millimeters with UBM for all patients prior to the brachytherapy and at 4-month interval follow-up. Tumor regression was calculated as a percentage of decrease in the initial <i>D</i> and <i>B</i> values. <b><i>Results:</i></b> The study involved 10 patients with a mean age of 64.4 years (yr) (range 46–80 yr). <i>D</i> ranged from 1.82 to 5.5 mm (median 2.99 mm) and <i>B</i> from 2.32 to 12.38 mm (median 4.18 mm). The apical radiation dose in all patients was 100 Gy. The median follow-up was 42.02 months. Regression for <i>D</i> was 21.11 ± 13.66%, 31.09 ± 14.66%, and 34.92 ± 19.86% at 1st, 2nd, and 3rd year of the follow-up, respectively, while for <i>B</i> it was 21.58 ± 16.05%, 28.98 ± 17.71%, and 32.06 ± 18.96%, respectively. Tumor recurrence was documented in 2/10 patients. <b><i>Conclusion:</i></b> The major regression of AUM, treated with Ru-106 plaque types CCB and CCC, was documented in the first 2 years after brachytherapy in our study group. In the following years, only minimal regression was documented that warns of the need for close monitoring and active search for local recurrences.

scholarly journals Iceland screens, treats, or prevents multiple myeloma (iStopMM): a population-based screening study for monoclonal gammopathy of undetermined significance and randomized controlled trial of follow-up strategies

2021 ◽  
Vol 11 (5) ◽  
Author(s):  
Sæmundur Rögnvaldsson ◽  
Thorvardur Jon Love ◽  
Sigrun Thorsteinsdottir ◽  
Elín Ruth Reed ◽  
Jón Þórir Óskarsson ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Early Treatment ◽  
Monoclonal Gammopathy ◽  
Controlled Trial ◽  
Participation Rate ◽  
Population Based ◽  
Early Therapy ◽  
Screening Study ◽  
Undetermined Significance

AbstractMonoclonal gammopathy of undetermined significance (MGUS) precedes multiple myeloma (MM). Population-based screening for MGUS could identify candidates for early treatment in MM. Here we describe the Iceland Screens, Treats, or Prevents Multiple Myeloma study (iStopMM), the first population-based screening study for MGUS including a randomized trial of follow-up strategies. Icelandic residents born before 1976 were offered participation. Blood samples are collected alongside blood sampling in the Icelandic healthcare system. Participants with MGUS are randomized to three study arms. Arm 1 is not contacted, arm 2 follows current guidelines, and arm 3 follows a more intensive strategy. Participants who progress are offered early treatment. Samples are collected longitudinally from arms 2 and 3 for the study biobank. All participants repeatedly answer questionnaires on various exposures and outcomes including quality of life and psychiatric health. National registries on health are cross-linked to all participants. Of the 148,704 individuals in the target population, 80 759 (54.3%) provided informed consent for participation. With a very high participation rate, the data from the iStopMM study will answer important questions on MGUS, including potentials harms and benefits of screening. The study can lead to a paradigm shift in MM therapy towards screening and early therapy.

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