The effectiveness and safety of palbociclib and ribociclib in stage IV HR+/HER-2 negative breast cancer: a nationwide real world comparative retrospective cohort study

Introduction: Palbociclib and ribociclib are indicated in the first-line treatment of hormonal receptor-positive HER-2 negative (HR+/HER2- negative) advanced breast cancer. Although randomized-controlled trials (RCTs) proved their clinical efficacy, there are no observational studies yet to validate the clinical findings in the real-world. Therefore, this study aimed to evaluate and compare the clinical effectiveness and safety profiles of palbociclib and ribociclib in Qatar. Materials and methods: A retrospective observational study was conducted on HR+/HER-2-negative stage-IV breast cancer patients receiving palbociclib or ribociclib in the state of Qatar. Clinical data were collected from the National Center for Cancer Care and Research (NCCCR) in Qatar using Cerner. Primary outcomes were progression-free-survival (PFS) and overall-survival (OS) generated by Kaplan-Meier curves. Moreover, safety profiles of both two treatments were evaluated. Results: The data from 108 patients were included in the final analysis. There was no statistically significant difference in PFS between the palbociclib and ribociclib groups; PFS was 17.85 versus 13.55 months, respectively(p> 0.05). Similarly, there was no statistically significant difference in OS between the two medications, 29.82 versus 31.72 months, respectively(p>0.05). Adverse events were similar between the two groups. Neutropenia was the most common side effect in the study population accounting for 59.3% of the patients. Conclusions: Therefore, both treatments have similar efficacy and safety profiles. Further research on a larger-scale population and longer follow-up period is recommeneded.The author(s) declare financial support was received for the research, authorship, and/or publication of this article. Hamad Medical Corporation.Scopu

Pill-burden and its association with treatment burden among patients with advanced stages of chronic kidney disease

Introduction: Chronic kidney disease (CKD) is associated with multimorbidity and high treatment burden. Pill-burden is one component of the overall treatment burden. However, little is known about its magnitude and contribution to the overall treatment burden among patients with advanced stages of CKD. This study aimed to quantify the magnitude of pill-burden in dialysis-dependent vs. non-dialysis-dependent advanced-stage CKD patients and its association with treatment burden. Methods: This was a cross-sectional study for the assessment of pill-burden and treatment burden among non-dialysis and hemodialysis (HD)-dependent CKD patients. Pill-burden was quantified as "number of pills/patient/week" through electronic medical record, while treatment burden was assessed using the "Treatment Burden Questionnaire (TBQ)". Furthermore, oral and parenteral medication burden was also quantified. Data were analyzed using both descriptive and inferential analysis, including Mann - Whitney U test and two-way between groups analysis of variance (ANOVA). Results: Among the 280 patients included in the analysis, the median (IQR) number of prescribed chronic medications was 12 (5.7) oral and 3 (2) parenteral medications. The median (IQR) pill-burden was 112 (55) pills/week. HD patients experienced higher pill-burden than non-dialysis patients [122 (61) vs. 109 (33) pills/week]; however, this difference did not reach statistical significance (p = 0.81). The most commonly prescribed oral medications were vitamin D (90.4%), sevelamer carbonate (65%), cinacalcet (67.5%), and statins (67.1%). Overall, patients who had high pill-burden (≥112 pills/week) had significantly higher perceived treatment burden compared to low pill-burden patients (<112 pills/week) [47(36.2) vs. 38.5(36.7); p = 0.0085]. However, two-way ANOVA showed that dialysis status is the significant contributor to the treatment-burden in the high overall pill-burden group (p < 0.01), the high oral-medication-burden group (p < 0.01), and the high parenteral-medication-burden group (p = 0.004). Conclusions: Patients with advanced CKD experienced a high pill-burden, which increases the treatment burden; however, the dialysis status of the patient is the main factor affecting the overall treatment burden. Future intervention studies should target this population with an aim to reduce polypharmacy, pill-burden, and treatment burden, which may ultimately improve CKD patients' quality of life.The authors would like to acknowledge Qatar University for the funding provided for the study. This manuscript is part of an MSc dissertation submitted to Qatar University and related abstracts were presented and published as part of the 56th European Renal Association - European Dialysis and Transplant Association (ERA-EDTA) Congress, Budapest, Hungary, 13-16 June 2019. The study received funding from Qatar University (grant number: QUCG- CPH-22/23-592 and QUST-CPH-SPR/2017-19). The content of this paper is the sole responsibility of the authors.Scopu

CLINICAL AND PHARMACOECONOMIC ANALYSES OF CDK4/6 INHIBITORS USE IN STAGE IV BREAST CANCER FEMALES IN THE STATE OF QATAR: A COMPARATIVE RETROSPECTIVE OBSERVATIONAL STUDY WITH COST-EFFECTIVENESS AND COST-UTILITY ANALYSES

Introduction: Cyclin-dependent kinase 4 and 6 (CDK4/6) inhibitors are indicated in the first-line treatment of hormonal receptor positive and HER-2 negative (HR+/HER2- negative) advanced breast cancer. Although phase III randomized controlled trials (RCTs) proved their clinical efficacy, they can increase healthcare expenditure. To date, there are no observational studies to validate the clinical findings of the existed RCTs. In addition, only a few pharmacoeconomic evaluations were published regarding the two common CDK4/6 inhibiting agents, palbociclib and ribociclib to evaluate their financial burden. Objectives: To evaluate the clinical efficacy of palbociclib and ribociclib in the local settings in Qatar. Moreover, to conduct a thorough pharmacoeconomic analysis for the two medications and compare them in terms of their cost-effectiveness and cost-utility. Methodology: A retrospective observational study on HR+/HER-2 negative stage IV breast cancer patients receiving palbociclib or ribociclib in Qatar was conducted, followed by a comparative pharmacoeconomic analysis. Clinical data were collected from the National Center for Cancer Care and Research (NCCCR) from January 2017 to December 2019 using Cerner system. The primary outcomes were progression-free survival (PFS) and overall-survival (OS) generated by Kaplan Meier curves. Safety profiles of both of the two medications were also evaluated. Then, a thorough cost- analysis was conducted by accounting methodology to summarize the overall cost of the treatment strategies of palbociclib and ribociclib. Costs were obtained from the department of accounting and finance in the NCCCR. To evaluate the long-term costs and effectiveness, a 10-year within-cycle corrected Markov's model was developed. The Markov's model consisted of three main health states: progression-free (PFS), progressed disease (PD), and death. Costs were summarized from the cost-analysis, transition probabilities were calculated from individual patient data obtained in the clinical phase, and utilities were summarized from the published literature. Incremental cost-effectiveness ratio (ICER) and incremental cost-utility ratio (ICUR) were compared to a three gross-domestic-product (3 GDP) per capita. Deterministic and probabilistic sensitivity analyses were carried out. Modeling was conducted via TreeAge Pro software. Results: There was no statistically significant difference between the palbociclib and ribociclib groups in terms of PFS; median PFS time was 17.85 versus 13.55 months respectively; p> 0.05. Similarly, there was no statistically significant difference in terms of OS between the two medications 29.82 versus 31.72 months; p>0.05. For the pharmacoeconomic analysis, ribociclib dominated palbociclib in yielding an ICER of -83,090.88 QAR per life year gained, and ICUR of -31,868.06 QAR per quality-adjusted life year gained. The results remained robust in all cases of the deterministic sensitivity analyses suggesting ribociclib is more cost-effective than palbociclib. Taking all combined uncertainties into account, the overall confidence in the base-case conclusion was around 60%. Conclusions: Both treatment strategies have similar efficacy and safety profiles. Nonetheless, ribociclib is a more cost-effective option than palbociclib based on the base-case results and based on the addressed uncertainties related to the model inputs

Cost-Effectiveness and Cost-Utility of Palbociclib versus Ribociclib in Women with Stage IV Breast Cancer: A Real-World Data Evaluation

Palbociclib and ribociclib are indicated in the first-line treatment of hormonal-receptor-positive HER-2 negative (HR+/HER-2 negative) advanced breast cancer. Despite their clinical benefit, they can increase healthcare expenditure. Yet, there are no comparative pharmacoeconomic evaluations for them in developing countries, the Middle East, or Gulf countries. This study compared the cost-effectiveness of palbociclib and ribociclib in Qatar. A 10-year within-cycle-corrected Markov&rsquo;s model was developed using TreeAge Pro&reg; software. The model consisted of three main health states: progression-free (PFS), progressed-disease (PD), and death. Costs were obtained from the actual hospital settings, transition probabilities were calculated from individual-patient data, and utilities were summarized from the published literature. The incremental cost-effectiveness ratio (ICER) and the incremental cost-utility ratio (ICUR) were calculated and compared to three gross-domestic-products per capita. Deterministic and probabilistic sensitivity analyses were performed. Ribociclib dominated palbociclib in terms of costs, life-years gained, and quality-adjusted life-years gained. The conclusions remained robust in the different cases of the deterministic sensitivity analyses. Taking all combined uncertainties into account, the confidence in the base-case conclusion was approximately 60%. Therefore, in HR+/HER-2 negative stage IV breast cancer patients, the use of ribociclib is considered cost-saving compared to palbociclib

Pill-burden and its association with treatment burden among patients with advanced stages of chronic kidney disease

Introduction: Chronic kidney disease (CKD) is associated with multimorbidity and high treatment burden. Pill-burden is one component of the overall treatment burden. However, little is known about its magnitude and contribution to the overall treatment burden among patients with advanced stages of CKD. This study aimed to quantify the magnitude of pill-burden in dialysis-dependent vs. non-dialysis-dependent advanced-stage CKD patients and its association with treatment burden. Methods: This was a cross-sectional study for the assessment of pill-burden and treatment burden among non-dialysis and hemodialysis (HD)-dependent CKD patients. Pill-burden was quantified as “number of pills/patient/week” through electronic medical record, while treatment burden was assessed using the “Treatment Burden Questionnaire (TBQ)”. Furthermore, oral and parenteral medication burden was also quantified. Data were analyzed using both descriptive and inferential analysis, including Mann – Whitney U test and two-way between groups analysis of variance (ANOVA). Results: Among the 280 patients included in the analysis, the median (IQR) number of prescribed chronic medications was 12 (5.7) oral and 3 (2) parenteral medications. The median (IQR) pill-burden was 112 (55) pills/week. HD patients experienced higher pill-burden than non-dialysis patients [122 (61) vs. 109 (33) pills/week]; however, this difference did not reach statistical significance (p = 0.81). The most commonly prescribed oral medications were vitamin D (90.4%), sevelamer carbonate (65%), cinacalcet (67.5%), and statins (67.1%). Overall, patients who had high pill-burden (≥112 pills/week) had significantly higher perceived treatment burden compared to low pill-burden patients (<112 pills/week) [47(36.2) vs. 38.5(36.7); p = 0.0085]. However, two-way ANOVA showed that dialysis status is the significant contributor to the treatment-burden in the high overall pill-burden group (p < 0.01), the high oral-medication-burden group (p < 0.01), and the high parenteral-medication-burden group (p = 0.004). Conclusions: Patients with advanced CKD experienced a high pill-burden, which increases the treatment burden; however, the dialysis status of the patient is the main factor affecting the overall treatment burden. Future intervention studies should target this population with an aim to reduce polypharmacy, pill-burden, and treatment burden, which may ultimately improve CKD patients’ quality of life