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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.

PurposeTo assess and describe patient-reported outcomes (PROs) in women with locally advanced/unresectable or metastatic breast cancer (aBC/mBC) with hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR + /HER2 −) status receiving palbociclib combination therapy in a US real-world setting.MethodsA prospective, noninterventional, multicenter longitudinal study was conducted in US patients initiating treatment with palbociclib combination therapy for HR + /HER2 − aBC/mBC. PRO data (SF-12; CES-D-10; mood; pain; fatigue; interference of aBC/mBC or its treatment on family life, social life, physical activity, energy, and productivity; overall health rating; and quality of life [QOL]) were collected via a custom-developed mobile application at daily, weekly, and cycle-based intervals. Patient medical information (demographics, clinical characteristics, treatment information, and adverse events) was collected from medical records at baseline and at the end of the 6-month follow-up period.ResultsPatients’ general health status (SF-12) remained consistent throughout treatment and was generally consistent with published norms for individuals diagnosed with cancer. The presence of depression (CES-D-10) was low and did not change substantially over time. Mean pain and fatigue scores using an 11-point numeric rating scale were low and remained stable. Patients, on average, reported neutral or positive moods. Patient-reported QOL and overall health was primarily “Good,” “Very good,” or “Excellent.” Findings were consistent regardless of patient experience with neutropenia.ConclusionsPatients treated with palbociclib, on average, reported consistently low levels of pain and fatigue as well as good QOL and overall health that remained stable throughout the first 6 months of treatment regardless of episodes of neutropenia.

Purpose To evaluate the influence of recall periods on the assessment of physical function, we compared, in cancer and general population samples, the standard administration of PROMIS Physical Function items without a recall period to administrations with 24-hour and 7-day recall periods. Methods We administered 31 items from the PROMIS Physical Function v2.0 item bank to 2400 respondents ( n  = 1001 with cancer; n  = 1399 from the general population). Respondents were randomly assigned to one of three recall conditions (no recall, 24-hours, or 7-days) and one of two “reminder” conditions (with recall periods presented only at the start of the survey or with every item). We assessed items for potential differential item functioning (DIF) by recall time period. We then tested recall and reminder effects with analysis of variance controlling for demographics, English fluency, and co-morbidities. Results Based on conservative pre-set criteria, no items were flagged for recall time period-related DIF. Using analysis of variance, each condition was compared to the standard PROMIS administration for Physical Function (no recall period). There was no evidence of significant differences among groups in the cancer sample. In the general population sample, only the 24-hour recall condition with reminders was significantly different from the “no recall” PROMIS standard. At the item level, for both samples, the number of items with non-trivial effect size differences across conditions was minimal. Conclusions Compared to no recall, the use of a recall period has little to no effect upon PROMIS physical function responses or scores. We recommend that PROMIS Physical Function be administered with the standard PROMIS “no recall” period.

Women with hormone receptor (HR)-positive early-stage breast cancer (BC) have five-year survival rates of >90% but remain at serious risk for developing distant metastases beyond five years from diagnosis. This retrospective cohort study used data from the Surveillance, Epidemiology, and End Results (SEER) registries to examine associations between distant recurrence-free interval (DRFI) and risk of BC-specific mortality following distant relapse. The analysis includes 1,057 women with second primary stage IV BC who were initially diagnosed with AJCC stages I–III HR-positive BC between 1990 and 2016. Overall, 65% of women had a preceding DRFI of ≥5 years. Five-year BC-specific survival following development of distant recurrence was 52% for women with DRFI ≥ 5 years compared to 31% in women with DRFI of < 5 years. In multivariable analyses, risks of cancer-specific mortality following distant recurrence were lower in women with DRFI of 5 years or more (subdistribution hazard ratio = 0.72, 95% CI 0.58–0.89, p = 0.002). The results of this study may inform patient-clinician discussions surrounding prognosis and treatment selection among HR-positive patients who develop a distant recurrence of disease.

This study was a retrospective chart review performed to examine and describe physician practice patterns in managing attention deficit/hyperactivity disorder (ADHD) across Europe. Physicians treating ADHD in the UK, France, Germany, Italy, the Netherlands and Spain were recruited. Each physician abstracted medical records of five patients (aged 6–17 years at time of review) with a documented diagnosis of ADHD made between January 2004 and June 2007. Data provided by the physician via the abstraction included (a) physician characteristics, (b) patient characteristics, (c) ADHD diagnosis and (d) ADHD outcomes (adherence, symptom control and satisfaction). A total of 779 patients met study inclusion criteria. In the overall population, patients’ mean (SD) age at time of diagnosis was 8.9 (2.6) years. The predominant treatment choice was long-acting methylphenidate, which was prescribed to more than 56 % of patients. According to physicians, only 30.8 % of patients showed ‘complete symptom control’ on current treatment and only 31.8 % of physicians reported being ‘very satisfied’ with their patients’ current treatment. Physicians’ assessments of complete symptom control and physician satisfaction with treatment were low, indicating unmet needs with current ADHD management in Europe.

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.

We conducted a retrospective medical record review of 1,063 patients to examine patient characteristics and treatment patterns among adults with Philadelphia chromosome and/or BCR–ABL-positive chronic-phase chronic myeloid leukemia in the USA, UK, Germany, and Japan. Patients who were included received first-line treatment with imatinib between 1 January 2005 and 31 December 2009 and were not enrolled in a randomized clinical trial between first-line treatment initiation and end of recorded follow-up. The average age was 55 years; 60% were male. On average, patients were initiated on imatinib 2.7 months post-diagnosis and received therapy for 22 months [19 months (USA) to 25 months (Japan)], at a median dose of 400 mg/day. Thirteen percent [8% (Japan) to 16% (UK)] had dose escalation to a median 800 mg/day. Twenty-nine percent discontinued imatinib, primarily due to resistance or disease progression. Among 148 patients receiving second-line dasatinib and 113 receiving nilotinib, a greater proportion in the USA and Germany received nilotinib (54% in each country), while fewer patients in Japan and the UK received nilotinib (47 and 17%, respectively). On average, patients had second-line therapy initiated 25 months post-diagnosis and received treatment for 11 months (dasatinib) or 8 months (nilotinib). Patients initiating dasatinib had more advanced disease than those initiating nilotinib.

This study describes health-related quality of life (HRQoL) among older Medicare beneficiaries with hormone receptor-positive (HR+) early breast cancer (eBC). Women aged ≥65 years diagnosed with stage I-III HR+ eBC between 1997 and 2014 using the Surveillance, Epidemiology, and End Results Medicare Health Outcomes Survey Data Resource were included. HRQoL was measured using the Short Form Health Survey including physical/mental component summary (PCS/MCS) scores and subscales. Patient surveys  ≤ 24 months post-diagnosis were matched to non-cancer controls. Mean differences in HRQoL were compared using analysis of covariance. Among 1880 HR+ eBC patients versus 5640 matched non-cancer controls, eBC patients surveyed  ≤ 6 months post-diagnosis (n  =  530) scored lower on component scores (PCS mean difference  =  1.6 [95%CI: 0.6-2.6]; MCS mean difference  =  2.0 [95%CI: 1.0-3.0]) and multiple subscales. Among women surveyed 19 to 24 months post-diagnosis (n  =  402), mean differences in HRQoL were modest (PCS: 1.2 [95%CI: 0.1-2.4]; MCS: −1.5 [95%CI: −2.7 to −0.3]). Most differences in HRQoL following diagnosis of eBC did not indicate statistical significance or minimally important difference, emphasizing that preservation of HRQoL is an important and realistic goal among patients with eBC.

Background Observational cohort study to assess the association between adherence to oral 5-aminosalicylates (5-ASAs) and all-cause costs and health care utilization among patients with active ulcerative colitis (UC) in the United States. Methods Retrospective analysis of insurance claims from June 1997 to August 2006 in the LifeLink Database. Patient criteria: aged 18 or older with one or more claim(s) between June 1997 and August 2005 for a UC diagnosis and at least one oral 5-ASA prescription on or after the first observed UC diagnosis; continuous enrollment for at least 6 months prior to and 12 months following 5-ASA initiation (index date). As a proxy for active disease, patients needed to have at least two UC-specific non-pharmacy claims, at least 30 days of 5-ASA treatment and at least one corticosteroid prescription within the 12-month post-index period. Cumulative exposure to oral 5-ASAs over the 12-month period was calculated using the medication possession ratio (MPR). Patients with an MPR of at least 0.80 were classified as adherent. All-cause medical and pharmacy resource utilization and costs were computed over the 12-month post-index period and compared between adherent and nonadherent patients. Results 1,693 UC patients met study inclusion criteria: 72% were nonadherent to 5-ASA treatment (n = 1,217) and 28% were adherent (n = 476) in the 12-month study period. Compared with nonadherent patients, adherent patients had 31% fewer hospitalizations ( P  = 0.0025) and 34% fewer emergency department admissions ( P  = 0.0016). Adherent patients had 25% more pharmacy prescriptions overall ( P <0.0001) and 71% more UC-related pharmacy prescriptions ( P <0.0001) than did nonadherent patients. Total all-cause health care utilization was 1.13 times higher for adherent patients than for nonadherent patients ( P  = 0.0002). After adjusting for covariates, total all-cause costs were 29% higher for nonadherent patients than for adherent patients (mean [95% confidence interval]: $13,465 [$13,094, $13,835] vs $17,339 [$17,033, $17,645]). Conclusions Approximately three-quarters of patients with active UC were not adherent with their prescribed doses of oral 5-ASA. Nonadherence was associated with higher total all-cause costs. The key driver of decreased costs among adherent patients was inpatient hospitalizations, which more than offset these patients’ expected higher pharmacy costs.

We conducted a review of patient medical records to assess treatment response patterns and prognostic indicators of response among chronic myeloid leukemia (CML) patients in the United States, the United Kingdom, Germany, and Japan. All 1,063 patients selected met the following inclusion criteria: aged 18 or older and in chronic phase at the time of diagnosis, Philadelphia chromosome and/or BCR-ABL positive, received first-line treatment with imatinib, and not enrolled in a randomized clinical trial during the period of retrospective review. Multivariable logistic regression models were used to evaluate prognostic indicators of complete hematological response (CHR), complete cytogenetic response (CCyR), and complete or major molecular response (C/MMR). Among patients treated with first-line imatinib, CHR at three months, CCyR at 12 months, and C/MMR at 18 months were observed in 53, 53.1, and 57.8 % of patients, respectively. Among patients treated with second-line dasatinib or nilotinib, CHR was achieved at three months in 49 and 42 %, CCyR at 12 months in 32 and 23 %, and MMR at 18 months in 30.5 and 26.1 % of patients, respectively. Prognostic indicators of first-line response included age, race, and Sokal score. For second-line treatment, duration of first-line hematological response and choice of drug used were also significant.