Patient reported disease-specific quality of life and symptom severity in systemic mastocytosis

BACKGROUND: Presently, no validated data exists on symptom severity and disease-specific quality of life (QoL) for mastocytosis patients. Simultaneously, clinical trials and drug application processes increasingly mandate reporting patients' perspectives on symptoms and QoL. We report on the development and validation of the mastocytosis quality of life questionnaire (MQLQ) and the mastocytosis symptom assessment form (MSAF). METHODS: Both outcome measures were developed in a standardised stepwise method, starting with the identification of items in focus groups (n= 12), item reduction and subsequent cross-sectional validation in a 63% female cohort of 164 adult indolent systemic mastocytosis patients. RESULTS: The MSAF reveals that fatigue is the severest mastocytosis symptom while the MQLQ indicates that fear of anaphylaxis most impacts QoL. Cross-sectional validity was assessed by correlating both individual domains and the total scores of the MQLQ and MSAF to independent measures of mastocytosis. The total scores of both the MQLQ (P< 0.001; Spearman's r: 0.568) and the MSAF (P< 0.001; Spearman's r: 0.559) correlated significantly with the consensus on physician scored mediator symptoms. The MQLQ domains displayed a high internal consistency (Cronbach's alpha: 0.841-0.958) and the domains "bones", "skin symptoms" and "anaphylaxis" differed significantly between patients with and without osteoporosis, urticaria pigmentosa or anaphylaxis respectively (P<0.001). CONCLUSIONS: The MQLQ is the first disease-specific QoL questionnaire for mastocytosis and is complemented by the MSAF, a short and convenient symptom scoring form. Both patient-reported outcome measures are valid, reliable and discriminate between patients with different disease characteristics making them useful instruments for clinical research. This article is protected by copyright. All rights reserved

An optically transparent thin-layer electrochemical cell for the study of vibrational circular dichroism of chiral redox-active molecules

An optically transparent thin-layer electrochemical (OTTLE) cell with a locally extended optical path has been developed in order to perform vibrational circular dichroism (VCD) spectroscopy on chiral molecules prepared in specific oxidation states by means of electrochemical reduction or oxidation. The new design of the electrochemical cell successfully addresses the technical challenges involved in achieving sufficient infrared absorption. The VCD-OTTLE cell proves to be a valuable tool for the investigation of chiral redox-active molecules

Clinical Impact of Skin Lesions in Mastocytosis: A Multicenter Study of the European Competence Network on Mastocytosis

Mastocytosis is a rare neoplasm characterized by the expansion and accumulation of mast cells in various organ systems. Systemic mastocytosis (SM) may or may not present with cutaneous lesions. To examine the frequency and clinical impact of cutaneous involvement, data on 1,510 patients with mastocytosis collected in the registry of the European Competence Network on Mastocytosis were analyzed. Cutaneous involvement was found in 1,195 of 1,510 patients (79.1%). Of these, 286 had cutaneous mastocytosis, and 721 had SM with skin involvement. Adult patients with skin involvement who did not have a bone marrow examination (n = 188) were defined as having mastocytosis in the skin. In 315 patients, SM without skin involvement was found. The percentage of cases with cutaneous involvement was higher in indolent SM (100%) and smoldering SM (87.9%) compared to aggressive SM (46.8%) or mast cell leukemia (38.5%). After a median follow-up of 5.6 years, no patient with cutaneous mastocytosis had died, but 2.6% of the patients with mastocytosis in the skin, 5.7% of the patients with SM with skin involvement, and 28.95% of the patients with SM without skin involvement had died. Overall survival was longer in patients with skin involvement (cutaneous mastocytosis and/or mastocytosis in the skin and/or SM with skin involvement) than in patients with SM without skin involvement (P &lt; 0.0001). These data argue for a thorough examination of both the skin and bone marrow in adult patients with mastocytosis