Validation of a patient-reported outcomes symptom measure for patients with nontransfusion-dependent thalassemia (NTDT-PRO© ).

This study demonstrates the quantitative characteristics of the first patient-reported outcome (PRO) tool developed for patients with nontransfusion-dependent ß-thalassemia (NTDT), the NTDT-PRO© . A multicenter validation study was performed over 24 weeks, involving 48 patients from Italy, Lebanon, Greece, and Thailand. Most patients were female (68.8%), with a median age of 34.5 years (range, 18-52); 66.7% were diagnosed with ß-thalassemia intermedia, and median time since diagnosis was 22 years (range, 0-43). The NTDT-PRO comprises 6 items across 2 domains (Tiredness/Weakness and Shortness of Breath [SoB]), and was valid and reliable, with good consistency. At baseline, most patients reported symptoms as present via the NTDT-PRO, and were highly compliant, ≥90% completing the NTDT-PRO tool. In a pairwise correlation analysis, all items were positively correlated. Correlations between NTDT-PRO and existing tools-36-Item Short Form Health Survey version 2 (SF-36v2) and Functional Assessment of Cancer Therapy-Anemia (FACT-An)-were assessed at weeks 1, 3, and 12; robust correlations were seen between SoB and SF-36v2-Vitality (rs = -0.53), and between SoB and Fact-An-Fatigue Experience (rs = -0.66) at week 1. Internal consistency was high for both Tiredness/Weakness (Cronbach alpha, 0.91) and SoB (Spearman-Brown coefficient, 0.78); intraclass correlation coefficients were high (Tiredness/Weakness, 0.88 and 0.97; SoB, 0.92 and 0.98), demonstrating stability. Further studies are required to fully support the validity of this tool, this study demonstrated the usefulness of the NTDT-PRO in the clinical setting and for longitudinal clinical research, particularly in trials where patient health-related quality of life is expected to change.

Health-Related Quality of Life in Patients with Metastatic Pancreatic Cancer.

PURPOSE: Due to its clinical course and often-late detection, many patients with metastatic pancreatic cancer (mPC) experience poor quality of life (QoL). This pilot project assessed real-world QoL in patients with mPC at different stages of treatment. METHODS: A cross-sectional survey was conducted in the following groups of patients with mPC: before initiation of first-line (1L) chemotherapy (no treatment); with partial response (PR) or stable disease (SD) upon receipt of ≥3 cycles of 1L nab-paclitaxel plus gemcitabine (nab-paclitaxel plus gemcitabine PR or SD); and with disease progression during ≥1L chemotherapy and not currently receiving nab-paclitaxel (≥1L PD). Eligible participants completed three QoL instruments, EORTC QLQ-C30, the pancreatic cancer module of EORTC QLQ-PAN26, and the EQ-5D, during their clinical visits at 14 clinics across the USA. RESULTS: Demographic characteristics were similar among groups (no treatment, n = 29; nab-paclitaxel plus gemcitabine PR or SD, n = 26; ≥1L PD, n = 17). Patients in the nab-paclitaxel plus gemcitabine PR or SD group had lower mean pain scores by EORTC-QLQ-C30 (27.6 vs 47.1; P = 0.02) and lower mean pancreatic pain scores by EORTC-QLQ-PAN26 (27.9 vs 45.4; P = 0.02) compared with the no treatment group. The groups did not differ significantly in QoL as measured by the EQ-5D. CONCLUSIONS: Patients who experienced PR or SD with 1L nab-paclitaxel plus gemcitabine had improved general and pancreatic pain scores and no clinically meaningful deterioration in QoL compared with patients who had not yet initiated chemotherapy.

Adherence to infectious diseases society of America guidelines for empiric therapy for patients with community-acquired pneumonia in a commercially insured cohort.

BACKGROUND: There is little published research addressing how the 2003 Infectious Diseases Society of America (IDSA) guidelines for empiric therapy of community-acquired pneumonia (CAP) are implemented in clinical practice. OBJECTIVE: This study was designed to describe antibiotic treatment patterns among patients with CAP treated in ambulatory settings in light of the IDSA guidelines. METHODS: Health insurance claims data from a large managed care organization with -30 million enrollees located in geographically diverse regions of the United States were analyzed. Patients > or =18 years of age with CAP who received a prescription for any antibiotic in an ambulatory setting during 2004 were identified via International Classification of Diseases, Ninth Revision, Clinical Modification codes for diagnosis (481-486). Recent antibiotic use was defined as receipt of any antibiotics <90 days before the date of diagnosis. Antibiotics were identified through National Drug Codes and from outpatient medical claims data with the use of J codes. Individuals were classified, per IDSA guidelines, as previously healthy without recent antibiotic use (group 1); previously healthy with recent antibiotic use (group 2); with comorbidities and without recent antibiotic use (group 3); and with comorbidities and recent antibiotic use (group 4). The guideline adherence was calculated using the number of patients receiving recommended treatment divided by the total number of patients in each group. RESULTS: Of 34,342 patients identified, 76.5% had no reported comorbidities. Among group-1 patients, 52.0% received the recommended empiric therapy (macrolide or doxycycline). In group-2 patients, 42.5% received the recommended therapy (respiratory quinolone alone or advanced-generation macrolide plus amoxicillin or amoxicillin-clavulanate). A high rate of compliance with recommended empiric therapy (advanced-generation macrolides or respiratory quinolones) was observed in group-3 patients (81.5%). In group-4 patients, 43.4% received the recommended therapy (respiratory quinolone or advanced-generation macrolide plus ss-lactam). Patients whose therapy was adherent with the guidelines had fewer respiratory-infection-related hospital admissions within 30 days after initiation of antibiotic treatment (overall, relative risk = 0.81 [95% CI, 0.71-0.94]). CONCLUSION: Although these data reflect a period shortly after the 2003 IDSA guidelines were published, they suggest that there is room for improvement with regard to choice of empiric antibiotic therapy among these patients with CAP treated in ambulatory settings.