Financial toxicity is prevalent and harmful to patients with plasma cell disorders.
Background: Female underrepresentation in oncology clinical trials can result in outcome disparities. We evaluated female participant representation in US oncology trials by intervention type, cancer site, and funding. Materials and Methods: Data were extracted from the publicly available Aggregate Analysis of ClinicalTrials.gov database. Initially, 270,172 studies were identified. Following the exclusion of trials using Medical Subject Heading terms, manual review, those with incomplete status, non-US location, sex-specific organ cancers, or lacking participant sex data, 1650 trials consisting of 240,776 participants remained. The primary outcome was participation to prevalence ratio (PPR): percent females among trial participants divided by percent females in the disease population per US Surveillance, Epidemiology, and End Results Program data. PPRs of 0.8-1.2 reflect proportional female representation. Results: Females represented 46.9% of participants (95% CI, 45.4-48.4); mean PPR for all trials was 0.912. Females were underrepresented in surgical (PPR 0.74) and other invasive (PPR 0.69) oncology trials. Among cancer sites, females were underrepresented in bladder (odds ratio [OR] 0.48, 95% CI 0.26-0.91, P =.02), head/neck (OR 0.44, 95% CI 0.29-0.68, P <.01), stomach (OR 0.40, 95% CI 0.23-0.70, P <.01), and esophageal (OR 0.40 95% CI 0.22-0.74, P <.01) trials. Hematologic (OR 1.78, 95% CI 1.09-1.82, P <.01) and pancreatic (OR 2.18, 95% CI 1.46-3.26, P <.01) trials had higher odds of proportional female representation. Industry-funded trials had greater odds of proportional female representation (OR 1.41, 95% CI 1.09-1.82, P =.01) than US government and academic-funded trials. Conclusions: Stakeholders should look to hematologic, pancreatic, and industry-funded cancer trials as exemplars of female participant representation and consider female representation when interpreting trial results.
The patient had a wide range of symptoms and comorbidities, resulting in a complicated differential diagnosis. Careful evaluation eventually led to a focus on sexually transmitted infection.
Allogeneic stem cell transplant (allo SCT) for multiple myeloma (MM) is potentially curative in some, while toxic in many others. We retrospectively analyzed 85 patients diagnosed with MM who underwent allo SCT as frontline or salvage therapy between 2000 and 2022 at Mayo Clinic Rochester and examined patient outcomes and prognostic markers. Overall survival (OS), progression free survival (PFS), treatment related mortality (TRM), and relapse rates (RR) were estimated using the Kaplan Meier method and competing risk models. Median follow-up was 11.5 years. Median OS and PFS were 1.7 and 0.71 years, respectively. Five-year OS and PFS were 22.2% and 15.1%, respectively. One-year TRM was 23.5%. Twelve patients demonstrated durable overall survival, living 10+ years beyond their allo SCT. This subgroup was more likely to have no or one prior auto SCT (p = 0.03) and to have been transplanted between 2000 and 2010 (p = 0.03). Outcomes were poor in this cohort with long follow-up, with few patients surviving 5 years or more, and most relapsing or dying within 2 years. We would expect better outcomes and tolerability with an expanded array of novel therapeutics and would prefer them to allo SCT.
The study aims to evaluate the risk factors and incidence of thromboembolic events among adult women with cancer who underwent controlled ovarian hyperstimulation (COH) for fertility preservation. Retrospective, descriptive cohort analysis of patient demographics, medical history, cancer type/treatment, laboratory values, thrombosis within 6 months of COH. 4 of 127 study participants experienced a venous thromboembolic event within 6 months of COH. The median time between oocyte aspiration and the event was 0.25 years (range = 0.10–0.50). The average age at time of event was 25.3 years (SD = 5.3). Three of four thrombotic patients had ovarian cancer, one had breast cancer. All had received surgery and chemotherapy for treatment. All underwent an antagonist cycle ovarian stimulation protocol — none developed ovarian hyperstimulation syndrome. The average anti-mullerian hormone level at the time of hyperstimulation in the thrombosis group was 1.6 (SD = 1.3), compared to 3.6 in the non-thrombosis group. The average max estradiol level reached during ovarian stimulation was 1281.3 (SD = 665.3) in the thrombosis group and 1839.1 (SD = 1513.9) in the non-thrombosis group. Thromboembolic events were not directly associated with mortality. Within this small descriptive study, the incidence of thromboembolic events in women with cancer undergoing COH for fertility preservation is high. Cancer may play a greater role than COH in thrombosis risk. Ovarian cancer patients who undergo ovarian stimulation may have an increased risk compared to other cancer types. These findings may inform future, prospective studies to determine the role of thromboprophylaxis.
Background Allogeneic stem cell transplant (allo-SCT) for treatment of multiple myeloma (MM) is controversial. For some, allo-SCT may drive treatment-resistant disease into remission through a graft vs myeloma effect. For others, it incurs considerable morbidity from opportunistic infections and graft vs host effects. Allo-SCT usage for MM has increased over the past 25 years despite mixed efficacy and the absence of clear treatment guidelines. We aimed to determine the natural history of patients who underwent an allo-SCT following an autologous stem cell transplant (ASCT) and identify characteristics associated with outcome. Methods We retrospectively analyzed 89 patients diagnosed with multiple myeloma who had received an allo-SCT following an ASCT between 2000 and 2022 at Mayo Clinic Rochester. Patient data were obtained from the Mayo Clinic Transplant Center Database and our institution's electronic medical records. Overall survival (OS) and progression free survival (PFS) were calculated as time from allo-SCT to death from any cause and first observation of relapse or death, respectively. OS and PFS rates were estimated using the Kaplan-Meier method. P < 0.05 was considered statistically significant. Results The cohort is characterized as follows: median patient age of 51.3 years, 71% male, 90% white/Caucasian. Indication for allo-SCT were 91.0% (N=81) relapsed/progressive disease, 9.0% (N=8) high risk in remission. Cytogenetic risk stratification at diagnosis was available for 94% of patients (N=84) with 63% (N=56) standard risk, 32% (N=28) high risk according to International Myeloma Working Group criteria. By conditioning regimen, 55% (N=49) underwent full myeloablative allo, 45% (N=40) non-myeloablative (mini)-allo. Median follow-up, OS, and PFS was 11.5, 1.7, and 0.8 years, respectively (Table 1). OS was 46% (N=41) at 2 years, 26% (N=21) at 4 years, and 16% (N=14) at 8 years (Figure 1). Outcome was not significantly impacted by age, sex, race, indication for allo-SCT, or myeloablative regimen. Patient cytogenetic risk at diagnosis approached, but did not reach, statistical significance (p=0.22) with overall survival of high-risk patients being 32% (N=9), 18% (N=5), and 7.1% (N=2) at 2, 4, and 8 years, respectively, compared to 52% (N=29), 27% (N=15), and 20% (N=11) over the same times in standard risk. PFS was 27% (N=24) at 2 years follow-up, 16% (N=14) at 4 years, and 7.9% (N=7) at 8 years (Figure 1). This did not vary significantly by age, race, indication for allo-SCT, cytogenetic risk, or conditioning regimen. Sex approached, but did not reach, statistical significance (p=0.16) with progression-free survival of females being 19% (N=5), 15% (N=4), and 7.7% (N=1) at 2, 4, and 8 years follow-up, respectively, compared to males at 30% (N=19), 16% (N=10), and 9.5% (N=6) over the same times. Discussion Long-term survival was achieved in less than 20% of patients who underwent allo-SCT following an ASCT. Nearly 75% of patients experienced disease progression or death by 2 years. This suggests minimal benefit to the use of allo-SCT as treatment for multiple myeloma outside of select patients. Novel therapeutics such as chimeric antigen receptor T cells (CAR-T) or bispecific T-cell engager (BiTE) should be considered as an alternative. Subsequent studies will further examine patient characteristics and associated outcomes to determine the population best suited for allo-SCT. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal
"A Rare Cause of Prevascular (Anterior) Mediastinal Mass." Annals of the American Thoracic Society, 19(5), pp. 850–853
Background: Advances in supportive care and novel therapies have driven improved outcomes for patients with Multiple myeloma (MM) and Light Chain Amyloidosis (AL). Unfortunately, this progress demands a heavy toll - higher patient costs. Financial toxicity (FT) is the adverse impact of cancer on a patient's financial well-being, contributing to increased distress, lower medication compliance, and even reduced survival. We aimed to evaluate MM and AL patients for financial toxicity over time to better understand its impact on various quality of life (QOL) domains.