Cognitive-behavioral methods seem to be appropriate for treating children with CAP.

A systematic approach to chronic recurrent abdominal pain in children and adolescents is key to ruling out organic diseases while avoiding unnecessary tests and treatments.

Hintergrund: Chronische Bauchschmerzen (CBS) sind im Kindesalter nicht nur weit verbreitet, sondern auch sehr stabil. Insbesondere psychosoziale Funktionseinschränkungen wie ein erhöhtes Stresserleben belasten die Kinder und deren Eltern und erschweren das Krankheitsmanagement; weitere komorbide Störungen können sich entwickeln. Dies legt nahe, dass eine Behandlung frühzeitig einsetzen und zudem auch die psychosozialen Aspekte mit berücksichtigen muss. Vor diesem Hintergrund wurde das kognitiv-behaviorale, kindzentrierte Gruppenprogramm «Stopp den Schmerz mit Happy-Pingu» entwickelt und evaluiert. Wie ist die psychosoziale Situation der betroffenen Kinder? Ist das kognitiv-behaviorale Programm in der Lage, psychosoziale Einschränkungen zu verbessern? Methodik: Das Programm besteht aus 6 Sitzungen für die Kinder sowie 1 Elternabend; die Trainingssitzungen fanden 1-mal wöchentlich statt. Im Rahmen einer randomisierten kontrollierten Studie wurde das Programm an 29 Kindern im Alter zwischen 6 und 12 Jahren auf seine Wirksamkeit hin überprüft. Die Evaluation erfolgte anhand eines Vergleiches zwischen Interventionsgruppe (IG) und Wartekontrollgruppe (WKG) zu den 3 Messzeitpunkten T1 (Prä), T2 (Post) und T3 (3-Monats-Follow-up). Ergebnisse: Bei den Kindern traten vor allem emotionale Probleme gehäuft auf. Die Akzeptanz des Programms war sehr hoch. Teilnehmer der IG erlebten im Vergleich zur WKG eine signifikante Verbesserung des Stresserlebens und der Coping-Strategien. Die Effektstärken sind überwiegend mittel bis hoch. Schlussfolgerungen: Multimodale kognitiv-behaviorale Ansätze scheinen geeignet, um Kinder mit CBS erfolgreich zu behandeln. Weitere kontrollierte Studien sollten unter anderem klären, welche Trainingselemente konkret für die Schmerzreduktion verantwortlich sind.

While the efficacy of cognitive-behavioral treatment (CBT) approaches for childhood functional abdominal pain (FAP) is well-established for child outcomes, only a few studies have reported on parent-specific outcomes. This randomized controlled pilot trial analyzed effects of a group CBT on maternal variables (i.e., pain-related behavior, worries and self-efficacy, as well as general psychosocial strain). Methods: The sample constituted of 15 mothers in the intervention group (IG) and 14 mothers in the waitlist control group (WLC). Outcome measures were assessed pre-treatment, post-treatment and at three months follow-up. Results: Analyses revealed significant, large changes in maladaptive maternal reactions related to the child’s abdominal pain in the IG compared to the WLC—i.e., reduced attention (d = 0.95), medical help-seeking (d = 0.92), worries (d = 1.03), as well as a significant increase in behaviors that encourage the child’s self-management (d = 1.03). In addition, maternal self-efficacy in dealing with a child’s pain significantly increased in the IG as well (d = 0.92). Treatment effects emerged post-treatment and could be maintained until three months follow-up. There were no effects on general self-efficacy and maternal quality of life. Conclusion: While these results are promising, and underline the efficacy of the CBT approach for both the child and mothers, further studies, including long-term follow-ups, are warranted.

Payers in European countries request studies with high levels of evidence for decision making also for rare diseases like haemophilia B (HB). The objective of the study was to determine the status quo of current studies in HB regarding the overall level of evidence generated. The methods used for performing the study were systematic literature research in EMBASE and MEDLINE, search terms 'HB' and 'factor IX' (FIX). The inclusion criteria were journal articles (JA), conference abstracts (CA), English language, published between January 2009 and March 2013, studies only; screening of titles, abstracts, full texts subsequently. ClinicalTrials.gov search: unpublished registered trials (RT) concerning HB or FIX. The analysis was performed on research topic, sponsor, recruitment status and study design. Screening of 1639 hits yielded 31 JA describing 35 studies, and 62 CA. FIX was subject of 21 studies (60.0%) and 29 CA (46.8%). Seven studies focused on various aspects of HB, six on haemophilia studies with separate HB data. Screening of 173 hits from ClinicalTrials.gov yielded 42 RT. Overall, 32 RT (76.2%) related to FIX. Measurement of health-related quality of life (HRQoL) was identified in none of these studies, four CA (6.5%), four RT (9.5%). Randomized study design was found in one study (2.9%), four RT (9.5%). Three studies (8.6%) and seven RT (16.7%) were prospective, observational and comparative. The majority of published clinical studies do not meet payers' expectations for evidence. Therefore, clinical investigation concepts addressing randomization, outcomes research including HRQoL and comparison of therapy options should be discussed. Refined statistical methods and exploitation of complementary real-life data sources may fill current evidence gaps concerning rare diseases.