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Palbociclib was the first cyclin-dependent kinase 4/6 inhibitor approved by the US Food and Drug Administration for use in combination with aromatase inhibitors (AIs) as initial endocrine-based therapy or with fulvestrant in postmenopausal women who previously received endocrine therapy based on data from randomized clinical trials. Real-world studies examining the effectiveness of palbociclib in large, diverse patient populations in routine clinical practice were needed. Ibrance Real World Insights (IRIS) was a retrospective medical record review study of women with confirmed hormone receptor–positive, HER2-negative advanced/metastatic breast cancer treated with palbociclib plus an AI or with palbociclib plus fulvestrant according to approved indications. Participating physicians reviewed medical records of up to 16 sequentially presenting patients, collecting demographic and clinical data. Outcomes included objective response rates, progression-free rates, and survival rates overall and in patients stratified according to age, race and ethnicity, Eastern Cooperative Oncology Group (ECOG) performance status (PS), disease-free interval, visceral disease, liver metastases, bone-only metastases, and previous lines of therapy. Data were abstracted by 417 physicians for 2954 patients in 13 countries; 1415 patients (47.9%) were ≥65 years of age, 369 patients (12.5%) had an ECOG PS ≥2 at initiation, and 835 patients (28.3%) were races other than White. The 12-month progression-free rate was 88% for palbociclib plus an AI and 79% for palbociclib plus fulvestrant; the 12-month survival rate was 96% in both groups. The objective response rates were 80% for palbociclib plus an AI and 75% for palbociclib plus fulvestrant. Palbociclib was similarly effective in most subgroups examined. Data from IRIS provide in-depth, real-world evidence for the use of palbociclib in a range of breast cancer populations in multiple countries. These data support the findings of the randomized PALOMA-2 and PALOMA-3 studies.

Introduction Vitiligo may negatively impact quality of life (QOL). This study aimed to classify patients on the basis of the impact of vitiligo on emotional/psychological and social functioning and describe patient characteristics and patient-reported outcome (PRO) responses for each profile. Methods Physician and patient surveys from the Vitiligo Disease Specific Programme in France, Germany, Italy, Spain, and the USA were analyzed. PROs were assessed using the Vitiligo Patient Priority Outcomes (ViPPO), Work Productivity Activity Index–Vitiligo, Vitiligo-Specific Quality-of-Life instrument, 5-level EuroQol 5-dimensional questionnaire (EQ-5D-5L), and Hospital Anxiety and Depression Scale questionnaires. Latent class analysis identified distinct classes on the basis of ViPPO response options. Results A three-class model was selected. Of 530 patients, 64.2%, 24.3%, and 11.5% were grouped into classes 1, 2, and 3, respectively. Class 3 reported the most emotional/psychological and social functioning impact; classes 1 and 2 reported mild and moderate impact, respectively. More patients in classes 1 and 2 had moderate physician-assessed disease; class 3 had a higher proportion of patients with moderate-to-severe disease; however, approximately one third had limited/mild physician-reported disease. Class 3 had the highest mean body surface area involvement and proportion of patients with face involvement ( n  = 30 [49.2%]). Across all 4 PRO measures, class 3 reported more severe impacts. Conclusions Generally, classes with more severe impacts had more severe physician-assessed disease and lesion involvement and greater work productivity and QOL impairment. Although patients in class 3 had severe psychosocial burden, a large proportion had mild physician-reported disease severity, suggesting that only physical symptoms were considered when assessing disease severity.

Introduction Alopecia areata (AA) is an autoimmune disease that causes scalp, face, and/or body hair loss. Recently, oral treatments with kinases inhibition became the first approved therapies for severe AA. An understanding of the use and effectiveness of traditional therapies in real-world treatment settings is needed to guide integration of novel therapies into the treatment paradigm. This study aimed to describe traditional treatment patterns, dermatologists’ reasons for therapy choice, and dermatologists’ satisfaction with disease control among patients with AA. Methods Data were drawn from the 2021–2022 Adelphi Real World AA Disease Specific Programme™, a cross-sectional survey of dermatologists and adult patients with AA, conducted in France, Germany, Italy, Spain, and the UK. For each patient, using data from patient consultation and medical records, dermatologists reported % scalp hair loss (SHL), characteristics of current and prior AA therapies, and satisfaction with disease control. Results Overall, 239 dermatologists provided data for 1720 patients with AA. Mean (SD) patient age was 35.8 (11.6) years, and 51% were male. Based on dermatologist perception, among patients with ≤ 10% SHL, 74% were experiencing mild AA, while ≥ 95% of patients with ≥ 50% SHL were experiencing severe/very severe AA. In patients with ≥ 50% SHL, the most common therapies received included systemic immunosuppressants (31%), topical corticosteroids (24%), and oral corticosteroids (24%). Among all patients who had switched therapies, 49%, 26%, and 24% switched because of worsening AA, lack of initial efficacy with prior treatment, and loss of response over time, respectively. Among those with SHL ≥ 50%, dermatologists reported satisfaction with current therapy in < 30% of patients. Conclusion Dermatologists reported low satisfaction with traditional AA therapies used in patients with extensive SHL, with some patients discontinuing treatment because of worsening disease. This suggests more effective treatments are needed for patients with severe AA.

Several adjuvant phase III trials are evaluating cyclin-dependent kinase 4/6 inhibitors (CDK4/6is) in combination with endocrine therapy (ET) in hormonal receptor positive (HR+)/human epidermal growth factor receptor 2 negative (HER2-) early-stage breast cancer (eBC). This study examines preferences for this combination regimen and ET alone among patients, oncologists, and payers in the United States. A web-based questionnaire, including a discrete choice experiment (DCE), was administered to patients, practicing oncologists, and payers. In the DCE, respondents selected between hypothetical treatment profiles with attributes associated with ET monotherapy and CDK4/6i + ET regimens. Each treatment alternative was defined by the following attributes: 5-year invasive disease-free survival (iDFS), nausea, diarrhea, neutropenia, alopecia, dosing schedule, and electrocardiogram (ECG) monitoring. Payers had the additional attribute of annual per-patient treatment cost. Hierarchical Bayesian models were used to estimate relative preference weights for each attribute-level and relative attribute importance. For patients (n=300) and oncologists (n=200), iDFS was most important (2 to 3 times more important than the next most important attribute), followed by neutropenia and diarrhea risks for patients and oncologists, respectively. Patients and oncologists required an improvement in iDFS of 8.0 and 5.6 percentage-points, respectively, to accept an increase in diarrhea risk from 11% to 81%. Payers (n=60) viewed annual per-patient cost as most important for treatment access decision-making, closely followed by iDFS. Payers required an improvement in iDFS of 21.8 percentage-points to accept an increase in cost from $5,100 to $149,400. Across all stakeholder groups, dosing schedule, alopecia risk, and ECG monitoring were perceived as least important. Patients, oncologists, and payers expect a large absolute risk reduction in efficacy to offset the potential risks and costs of adding a CDK4/6i to current standard of care. An open discussion between all stakeholders is necessary to ensure that decision-making, whether at patient- or system-level, is informed by preferences for novel treatments, like CDK4/6is.

Background The Severity of Alopecia Tool (SALT) is a clinician‐reported outcome measure of scalp hair loss in alopecia areata (AA). Objectives To characterise the magnitudes of change in SALT scores corresponding to meaningful treatment benefits from the patient's perspective. Methods Anchor‐based methods for the estimation of meaningful within‐patient change thresholds were applied to pooled data from a randomised, double‐blind trial of ritlecitinib. Anchors included a patient‐reported measure of change in AA severity, the Patient Global Impression of Change (PGI‐C) and three items comprising the Patient Satisfaction with Hair Growth (P‐Sat) questionnaire. After reviewing Pearson correlations between change‐from‐baseline SALT scores and each anchor to confirm adequate association, potential thresholds were computed as mean change‐from‐baseline SALT scores among patients who reported moderate improvement on the PGI‐C and/or moderate satisfaction on each of three P‐Sat items at week 24. Results Six hundred and fifty participants (86% adults, 14% adolescents) had mean (standard deviation) SALT scores of 90.6 (14.3) at baseline, suggesting a sample with primarily severe AA. Correlations between SALT change‐from‐baseline scores and the patient‐reported items supported their use as anchors. Estimates based on patients reporting moderate improvement in AA (n = 102) on the PGI‐C and those reporting moderate satisfaction on the P‐Sat item related to the amount of hair growth at week 24 (n = 122) were −42.2 (26.1) and −43.1 (26.8), respectively. Supportive estimates based on the remaining P‐Sat items were similar in magnitude. Conclusions Among patients with severe AA, SALT change‐from‐baseline scores of 42 or 43 represent meaningful improvements. While the achievement of low SALT scores of ≤10–≤20 have been used to characterise efficacy in clinical trials, the amount of change required to meet this endpoint far exceeds the estimates in this study. The treatment goals of individual patients must be considered when evaluating benefit in both clinical trials and clinical practice. Applying anchor‐based methods, we used pooled data from a randomised, double‐blind trial of ritlecitinib to estimate meaningful within‐patient change thresholds for the Severity of Alopecia Tool (SALT), a clinician‐reported measure of scalp hair loss in alopecia areata (AA). Based on patients reporting moderate improvement in AA on the Patient Global Impression of Change and reporting moderate satisfaction on the Patient Satisfaction with Hair Growth at Week 24, estimates of meaningful SALT change from baseline scores were 42 and 43, respectively.

PurposeAlopecia areata (AA) is an autoimmune-mediated inflammatory dermatological disease characterised by non-scarring hair loss affecting the scalp and sometimes other hair-bearing sites. This study aimed to elicit health state utility values (HSUVs) from the UK general population for AA using time trade off (TTO) interviews.MethodsVignette descriptions of health states defined by the extent of hair loss were developed (as well as one describing caregiver burden). These were developed using data from standardised patient reported outcome (PRO) measures, a literature review and qualitative interviews. Health states were defined based on the severity of alopecia tool (SALT), which assesses extensiveness of scalp hair loss. HSUVs were then elicited for each health state in TTO interviews with the UK public.ResultsOne caregiver and five patient health states were developed based on the literature review findings, clinical trial PRO (Hospital Anxiety and Depression Scale and Alopecia Areata Patient Priority Outcomes Questionnaire) data and qualitative interviews with patients (N = 11), clinical experts (N = 4) and caregivers of adolescents with AA (N = 10). These data showed a more severe impact among patients with more extensive hair loss. One hundred and twenty participants evaluated the vignettes in TTO interviews. Patient HSUVs ranged from 0.502 for the most extensive hair loss health state (SALT 50–100 + eyebrow and eyelash loss) to 0.919 (SALT 0–10) for the mildest health state. The caregiver HSUV was 0.882.ConclusionQuantitative and qualitative data sources were used to develop and validate vignettes describing different AA health states. Patient and caregiver HSUVs demonstrate a large impact associated with AA, especially for states defined by more extensive hair loss.

The goal of this study was to characterize health-related quality of life (HRQOL) among patients diagnosed with early-stage, hormone receptor–positive (HR+)/human epidermal growth factor receptor 2 negative (HER2–) breast cancer. A multinational (United States, Japan, France, Germany, Italy, Spain, and United Kingdom) study of patients diagnosed with stage I to III HR+/HER2– breast cancer, either receiving adjuvant treatment or under postadjuvant surveillance, was conducted between June and October 2019. Patients were identified by their consulting physician and invited to complete the Functional Assessment of Cancer Therapy–Breast (FACT-B) and the EQ-5D-5L pen and paper questionnaires. EQ-5D-5L index scores were derived by using available country-specific health state value sets, where available, and numerically compared with general population scores derived from published normative and population data. Descriptive summary statistics were reported for FACT-B, Functional Assessment of Cancer Therapy-General (FACT-G) (total and specific subscales), the EQ-5D index scores, and the EQ-VAS scores for each country. Results were stratified according to disease-free treatment status (active adjuvant treatment or postadjuvant surveillance), age (25–44, 45–54, 55–64, or ≥65 years), stage (I, II, or III), and menopausal status at the time of questionnaire completion (pre-/peri-menopausal or postmenopausal). Overall, 1110 patients completed the HRQOL questionnaires (mean age, 59 years; 79% active adjuvant treatment, and 21% under surveillance postadjuvant treatment at time of questionnaire administration; 31% stage I, 48% stage II, and 20% stage III at diagnosis). Of these, 1102 completed the FACT-B and 1083 completed the EQ-5D-5L questionnaires. The mean (SD) FACT-B total score was 99.0 (21.9). The mean FACT-G total score was 72.5 (17.8), which was comparable to the published normative score. The mean EQ-5D index and EQ-VAS scores for each country were similar to corresponding population means; EQ-5D index scores ranged from 0.842 (0.098) in Japan to 0.916 (0.109) in France, and EQ-VAS scores from 68.0 (18.4) in Germany to 78.6 (16.4) in the United States. In addition, mean scores were comparable between the active adjuvant treatment and postadjuvant surveillance groups for the FACT-B total (99.4 [22.5] and 97.7 [19.7], respectively), FACT-G total (72.8 [18.3] and 71.3 [16.0]), EQ-5D index score (0.868 [0.135] and 0.869 [0.142]), and EQ-VAS (74.9 [17.2] and 74.4 [16.1]). Patient-reported HRQOL among patients with HR+/HER2– early breast cancer who were disease-free was high, with reported scores comparable to normative scores. These results improve our understanding of HRQOL among patients with early disease and may facilitate future studies examining the potential impact of adjuvant treatment and disease recurrence, including metastasis.

Patient experience literature in early-stage breast cancer (eBC) is limited. This study used a mixed-methods approach to examine patient conversations from public online forums to identify and evaluate eBC-related themes. Among 60,000 eBC-related posts published September 2014–2019, text from a random subset of 15,000 posts was extracted and grouped into linguistically similar, mutually exclusive clusters using an advanced natural language processing (NLP) algorithm. Clusters were characterized using four quantitative metrics: betweenness centrality (linguistic similarity to other areas of the cluster network), sentiment (general attitude toward a topic), recency (average date of posts), and volume (total number of posts). This analysis represented 3906 unique users (67% and 33% obtained from cancer–specific and general health/nonhealth forums, respectively). Of the 27 clusters identified, most important were “discussing recurrence & progression,” “understanding diagnosis & prognosis,” and “understanding cancer, biomarkers, and treatments.” Several major themes related to recurrence risk, diagnosis, monitoring, and treatment were identified. Additional emphasis on communicating the disease recurrence risk and shared decision-making could strengthen patient-clinician partnerships.

The ALLEGRO phase 2b/3 study investigated the efficacy and safety of ritlecitinib in patients with alopecia areata (AA). To describe the impact of ritlecitinib on patient-reported hair loss using the Alopecia Areata Patient Priority Outcomes (AAPPO) instrument and evaluate the relationship between clinically meaningful hair regrowth and improvements in patient-reported impacts. In ALLEGRO-2b/3, patients aged ≥ 12 years with AA and ≥ 50% scalp hair loss received once-daily ritlecitinib 50 or 30 mg (± 4-week 200-mg daily loading dose), 10 mg, or placebo for 24 weeks and then continued ritlecitinib or switched from placebo to ritlecitinib 200/50 or 50 mg for 24 weeks. The AAPPO instrument evaluated improvement in hair loss, emotional symptoms (ES), and activity limitations (AL) from weeks 4 to 48 (secondary endpoint). Mean changes in ES and AL domain scores and individual items at weeks 24 and 48 were calculated for Severity of Alopecia Tool (SALT) score ≤ 20 responders and nonresponders (exploratory endpoint). Overall, 718 patients were randomized. At week 24, 5-36% of patients receiving ritlecitinib 10-200/50 mg reported improvement in scalp hair loss versus 9% receiving placebo. The results for eyebrow, eyelash, and body hair loss were similar. Mean change from baseline in ES and AL scores at weeks 24 and 48 was small and similar between groups. Mean change was larger for individual hair loss and ES items at weeks 24 and 48 in SALT score ≤ 20 responders versus nonresponders. The AAPPO instrument demonstrated the beneficial impact of ritlecitinib on patient-reported hair growth, which was consistent with improvements in clinician-reported outcomes. NCT03732807. INFOGRAPHIC.