Characteristics of the patients.

<p>Characteristics of the patients.</p

An Italian experience

Background: Haemophilia with inhibitors is a rare condition, which requires a substantial amount of economical and human resources representing one of the most challenging complications of haemophilia treatment. Quality of life and self-management abilities of patients are affected by this complication. In the Italian COHIBA Study haemophilia patients with inhibitors were asked about their treatment preferences, HRQoL and treatment satisfaction. Methods: HRQoL was evaluated with the Core Instrument of the haemophilia-specific Haem-A-QoL consisting of 25 items (allowing the comparison between children and adults), treatment satisfaction was assessed with the Hemo-Sat questionnaire (for adults and parents of haemophilic children) consisting of 34 items pertaining to 6 dimensions (ease & convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction). Results:In total 23 adult and 7 paediatric inhibitor patients, severely affected by haemophilia A, were enrolled from 9 Italian Haemophilia Centers. 56% received on-demand treatment, 22% prophylaxis and 22% ITI. Median age in adults was 40 years (17\u201360), in children 10 years (4\u201316). Patients and parents were mainly unsatisfied with haemophilia treatment in terms of 'efficacy' (MA= 56.25 \ub1 18.7; MP= 72.22 \ub1 11.7) and 'ease' (MA= 53.38\ub110.42; MP= 61.21\ub18.6). Adult patients showed main impairments in HRQoL in the dimensions 'sport', 'physical health' and 'future'. Whereas children were less impaired in their HRQoL than adult patients; areas of main impairments were 'sport', 'dealing' and 'view'. Conclusions: Adult patients were more impaired in their HRQoL compared to children, even though both were mainly limited in sports activities. Findings are important to understand specific problems of inhibitor patients and to improve their individual treatmen

Health-related quality-of-life in patients with newly diagnosed multiple myeloma in the FIRST trial: lenalidomide plus low-dose dexamethasone versus melphalan, prednisone, thalidomide.

We compared the health-related quality-of-life of patients with newly diagnosed multiple myeloma aged over 65 years or transplant-ineligible in the pivotal, phase III FIRST trial. Patients received: i) continuous lenalidomide and low-dose dexamethasone until disease progression; ii) fixed cycles of lenalidomide and low-dose dexamethasone for 18 months; or iii) fixed cycles of melphalan, prednisone, thalidomide for 18 months. Data were collected using the validated questionnaires (QLQ-MY20, QLQ-C30, and EQ-5D). The analysis focused on the EQ-5D utility value and six domains pre-selected for their perceived clinical relevance. Lenalidomide and low-dose dexamethasone, and melphalan, prednisone, thalidomide improved patients' health-related quality-of-life from baseline over the duration of the study across all pre-selected domains of the QLQ-C30 and EQ-5D. In the QLQ-MY20, lenalidomide and low-dose dexamethasone demonstrated a significantly greater reduction in the Disease Symptoms domain compared with melphalan, prednisone, thalidomide at Month 3, and significantly lower scores for QLQ-MY20 Side Effects of Treatment at all post-baseline assessments except Month 18. Linear mixed-model repeated-measures analyses confirmed the results observed in the cross-sectional analysis. Continuous lenalidomide and low-dose dexamethasone delays disease progression versus melphalan, prednisone, thalidomide and has been associated with a clinically meaningful improvement in health-related quality-of-life. These results further establish continuous lenalidomide and low-dose dexamethasone as a new standard of care for initial therapy of myeloma by demonstrating superior health-related quality-of-life during treatment, compared with melphalan, prednisone, thalidomide