Development of a core outcome domain set for clinical research on capillary malformations (the COSCAM project).

BACKGROUND: Due to a large variety in treatment outcomes reported in therapeutic trials and lacking patient-relevant outcomes, it is hard to adequately compare and improve current therapies for patients with capillary malformations (CMs). The Core Outcome Set for Capillary Malformations (COSCAM) project aims to develop a core outcome set (COS) for use in future CM trials, in which we will first develop a core outcome (sub)domain set (CDS). Here, we describe the methods for the development of a CDS and present the results of the first development stage. METHODS: The COSCAM project is carried out according to the recommendations of the Cochrane Skin Core OUtcomes Set INitiative (CS-COUSIN) and the Core Outcome Measures in Effectiveness Trials (COMET) initiative. During the first stage, we identified all potentially relevant outcome subdomains based on a systematic review, two focus group sessions and input from patient representatives of Dutch patient organizations and the COSCAM-founding group. In stage two, we will present the subdomains in a three-round e-Delphi study and online consensus meeting, in which CM patients, parents/caregivers and CM experts worldwide rate the importance of the proposed subdomains, hereby finalizing the core outcome (sub)domains of the CDS. RESULTS: A total of 67 potential outcome subdomains were included; sixteen were previously used in the literature, 20 were proposed by Dutch patients and their parents/caregivers (n = 13) in focus group sessions and 38 were suggested by the experts of the COSCAM-founding group. Seven were excluded because of overlap. CONCLUSION: The final CDS may serve as a minimum standard in future CM trials, thereby facilitating adequate comparison of treatment outcomes. After this CDS development, we will select appropriate outcome measurement instruments to measure the core outcome subdomains.

Port wine stain treatment outcomes have not improved over the past three decades.

BACKGROUND: Since the early '80s, the pulsed dye laser has been the standard treatment tool for non-invasive port wine stain (PWS) removal. In the last three decades, a considerable amount of research has been conducted to improve clinical outcomes, given that a fraction of PWS patients proved recalcitrant to laser treatment. Whether this research actually led to increased therapeutic efficacy has not been systematically investigated. OBJECTIVE: To analyse therapeutic efficacy in PWS patients globally from 1986 to date. METHODS: PubMed was searched for all available PWS trials. Studies with a quartile percentage improvement scale were included, analysed and plotted chronologically. Treatment and patient characteristics were extracted. A mean clearance per study was calculated and plotted. A 5-study simple moving average was co-plotted to portray the trend in mean clearance over time. The data were separately analysed for multiple treatment sessions in previously untreated patients. RESULTS: Sixty-five studies were included (24.3% of eligible studies) comprising 6207 PWS patients. Of all patients, 21% achieved 75-100% clearance. Although a few studies reported remarkably good outcomes in a subset of carefully selected patients, there was no upward trend over time in mean clearance. CONCLUSION: The efficacy of PWS therapy has not improved in the past decades, despite numerous technical innovations and pharmacological interventions. With an unwavering patient demand for better outcomes, the need for development and implementation of novel therapeutic strategies to clear all PWS is as valid today as it was 30 years ago.

Diagnostic features of malignancy-associated pseudoachalasia.

BACKGROUND: Pseudoachalasia is a condition in which clinical and manometric signs of achalasia are mimicked by another abnormality, most often a malignancy. AIM: To identify risk factors that suggest presence of malignancy-associated pseudoachalasia. METHODS: In this retrospective cohort study, achalasia patients newly diagnosed by manometry were included. Patients with a normal initial endoscopy, clinical and manometric signs of achalasia who were afterwards found to have an underlying malignant cause were classified as pseudoachalasia. Clinical and diagnostic findings were compared between malignant pseudoachalasia and achalasia. RESULTS: We included 333 achalasia patients [180 male, median age 50 (38-62)]. Malignant pseudoachalasia was diagnosed in 18 patients (5.4%). Patients with malignancy-associated pseudoachalasia were older at time of diagnosis [67 (54-71) vs. 49 (37-60) years], had a shorter duration of symptoms [6 (5-10) vs. 25 (11-60) months] and lost more weight [12 (9-17) vs. 5 (0-12) kg). In 61% of the pseudoachalasia patients, the oesophagogastric junction (OGJ) was difficult or impossible to pass during endoscopy, compared to 23% in achalasia. Age ≥55 years (OR 5.93), duration of symptoms ≤12 months (OR 14.5), weight loss ≥10 kg (OR 6.73) and difficulty passing the OGJ during endoscopy (OR 6.06) were associated with a higher risk of malignant pseudoachalasia. CONCLUSIONS: Advanced age, short duration of symptoms, considerable weight loss and difficulty in passing the OGJ during endoscopy, are risk factors that suggest potential malignancy-associated pseudoachalasia. To exclude pseudoachalasia, additional investigations are warranted when two or more risk factors are present.