Uitkomstenonderzoek dure geneesmiddelen. Pilot studies bortezomib en oxaliplatin: resultaten en methodologische aanbevelingen

In het kader van de NZa beleidsregels ‘Dure geneesmiddelen’ en ‘Weesgeneesmiddelen in academische ziekenhuizen’ beoordeelt het CVZ sinds 1 januari 2006 of dure geneesmiddelen tijdelijk kunnen worden opgenomen in deze beleidsregels en daarmee aanspraak maken op additionele financiering. Deze aanspraak is gekoppeld aan het verzamelen van gegevens in de klinische praktijk, het uitkomstenonderzoek. Op basis van het uitkomstenonderzoek vindt na 4 jaar een herbeoordeling plaats, waarin naast de therapeutische waarde en het kostenbeslag ook de kosteneffectiviteit wordt beoordeeld. Tot 2012 adviseerde het CVZ de NZa en VWS op basis van deze herbeoordeling over continuering van additionele financiering middels de NZa beleidsregels

Health related quality of life in patients with multiple myeloma undergoing a double transplantation

Objectives: To investigate the subjective well-being of patients with newly diagnosed multiple myeloma who were treated in a tandem transplantation programme. Methods: Fifty-one patients participated in the prospective, longitudinal questionnaire study. The EORTC QLQ-C30 and the EuroQol-5D were administered 2 wk after completion of vincristine, adriamycin and dexamethason/vincristine, adriamycin and methyl prednison (VAD/VAMP) chemotherapy, both at hospital discharge after treatment with high-dose melphalan (HDM) and 1 month after this hospital discharge, at hospital admission, at the day of hospital discharge for peripheral stem cell transplantation (PSCT) and at 6 and 12 months following discharge after PSCT. Results: Overall, patients' functioning improved during treatment and follow-up, with significant decreases shortly following PSCT. Shortly after HDM and PSCT, patients reported a considerable increase in levels of soreness in the mouth (+ 26/ + 36 points on a scale ranging form 0 to 100; P < 0.01), change of taste (+ 23/ + 21 points; P < 0.05/ NS), nausea/vomiting (+ 26

Quality of life of patients with chronic lymphocytic leukaemia in the Netherlands: results of a longitudinal multicentre study

Purpose: To describe the health-related quality of life (HRQoL) of an unselected population of patients with chronic lymphocytic leukaemia (CLL) including untreated patients. Methods: HRQoL was measured by the EORTC QLQ-C30 including the CLL16 module, EQ-5D, and VAS in an observational study over multiple years. All HRQoL measurements per patient were connected and analysed using area under the curve analysis over the entire study duration. The total patient group was compared with the general population, and three groups of CLL patients were described separately, i.e. patients without any active treatment (“watch and wait”), chlorambucil treatment only, and patients with other treatment(s). Results: HRQoL in the total group of CLL patients was compromised when compared with age- and gender-matched norm scores of the general population. CLL patients scored statistically worse on the VAS and utility score of the EQ-5D, all functioning scales of the EORTC QLQ-C30, and the symptoms of fatigue, dyspnoea, sleeping disturbance, appetite loss, and financial difficulties. In untreated patients, the HRQoL was slightly reduced. In all treatment stages, HRQoL was compromised considerably. Patients treated with chlorambucil only scored worse on the EORTC QLQ-C30 than patients who were treated with other treatments with regard to emotional functioning, cognitive functioning, bruises, uncomfortable stomach, and apathy. Conclusions: CLL patients differ most from the general population on role functioning, fatigue, concerns about future health, and having not enough energy. Once treatment is indicated, HRQoL becomes considerably compromised. This applies to all treatments, including chlorambucil, which is considered to be a mild treatment

Autologous Stem Cell Transplantation in elderly Acute Myeloid Leukemia

Treatment outcome in elderly Acute Myeloid Leukemia (AML) is still very disappointing. Although complete remission rate is around 50-60% the 2 years survival is only in the magnitude of 10-20%. This is mainly due to an overrepresentation of adverse prognostic factors present in elderly AML. As relapses emerge from residual disease present after chemotherapy, intensification of treatment could emerge as a rational strategy. Intensification of chemotherapy by increasing the dose of  anthracyclines or addition of gemtuzumab ozogamycin (Mylotarg) to standard chemotherapy indeed has proved to be of advantage in elderly AML. In younger AML autologous peripheral blood stem cell transplantation (AuPBSCT) as post remission treatment in comparison to intensive consolidation chemotherapy has been investigated in a few randomized studies. AuPBSCT showed reduced relapse rates with low non-relapse mortality rates. In elderly AML intensification by AuPBSCT also have been performed although randomized studies are lacking. Nevertheless, in the previous years various reports have suggested the potential utility of AuHSCT in AML of the elderly with encouraging results, albeit mostly in highly selected patients. Acceptable toxicity and a relatively low rate of transplant-related mortality has been notified. However relapses occurred  which, irrespective of age, still remains the major cause of treatment failure of AuHSCT in AML. In this review we summarize the experience of AuPBSCT in elderly AML