Efficacy of a Brief, Peer-Delivered Self-management Intervention for Patients With Rare Chronic Diseases

Abstract

IMPORTANCEPatients coping with rare diseases need psychosocial support.OBJECTIVE To evaluate the efficacy of a brief, transdiagnostic, peer-delivered intervention for patients with rare diseases in addition to care as usual (CAU) compared with CAU only. DESIGN, SETTING, AND PARTICIPANTSIn this 2-group randomized clinical trial conducted from October 5, 2017, to July 12, 2019, patients were recruited via specialized clinics and patient organizations across Germany and participated from home. The study included consecutive adult patients with neurofibromatosis type 1, Marfan syndrome, primary sclerosing cholangitis, and pulmonary arterial hypertension who have limited functionality because of the disease. Exclusion criteria were a life-threatening health status and ongoing psychotherapeutic treatment. Of 143 patients screened for eligibility with a semistructured telephone interview, 54 were excluded, and 89 were randomized: 45 patients were randomly allocated to the peer-delivered intervention group, and 44 to the control group; 87 patients (98%) completed the 6-month follow-up assessment. The analysis was performed using an intention-to-treat principle. Data cleansing and analysis were conducted between April 25, 2019, and February 13, 2020. INTERVENTIONSThe 6-week intervention consisted of a self-help book and telephone-based peer counseling in addition to CAU. The control group received CAU alone. Peer counselors received training, structured consultation guidelines, and supervision. MAIN OUTCOMES AND MEASURESThe primary outcome was acceptance of the disease as assessed using the Illness Cognition Questionnaire (ICQ; mean sum scores range from 0 to 18, with higher values representing more acceptance) 6 months after the intervention. Secondary outcomes included self-reported coping strategies (Health Education Impact Questionnaire), illness cognition (ICQ and Illness Perception Questionnaire), depression severity (Patient Health Questionnaire 9-item depression scale), anxiety severity (Generalized Anxiety Disorder Scale), quality of life (12-Item Short-Form Health Survey), and social support (Social Support Questionnaire). Outcomes were assessed before the intervention, after the intervention, and at a 6-month follow-up. RESULTSThe mean (SD) age of the 89 participating patients was 46.3 (14.9) years; 59 (66%) were women. There were no group differences regarding baseline variables. All patients allocated to the intervention group completed the intervention. Six months after the intervention, but not directly after completing the program, the intervention group had significantly higher rates of acceptance (ICQ) of the disease (primary outcome) compared with the CAU group. Mean (SD) baseline ICQ scores were 9.61 (3.79) in the control group and 9.86 (3.40) in the intervention group. Mean (SE) ICQ scores at 6 months were 10.32 (0.42) for the control group and 11.79 (0.42) for the intervention group, with a significant mean difference of −1.47 (95% CI, −2.63 to −0.31; P = .01). Several secondary outcomes, including d...

Psychosoziale Unterstützung von Menschen mit Colitis ulcerosa und Reizdarmsyndrom: eine Machbarkeitsstudie zu Bedarf, Fokus und Durchführbarkeit

“…to-peer Ansätze, welche den Austausch mit anderen Betroffenen fördern [13][14][15]. Neue Ansätze in der psychosozialen Unterstützung von Menschen mit chronischen Erkrankungen setzen die Optimierung von krankheitsbezogenen Erwartungen, zum Beispiel in Bezug auf den Verlauf der Erkrankung oder die Wirksamkeit und Verträglichkeit von Behandlungen, in den Fokus.…”

“…Darüber hinaus zielen zahlreiche Interventionen darauf ab, die Selbstmanagement-Fähigkeiten von Patient:innen zu verbessern 11 12 . Ebenfalls vielversprechend sind sogenannte Peer-to-peer Ansätze, welche den Austausch mit anderen Betroffenen fördern 13 14 15 . Neue Ansätze in der psychosozialen Unterstützung von Menschen mit chronischen Erkrankungen setzen die Optimierung von krankheitsbezogenen Erwartungen, zum Beispiel in Bezug auf den Verlauf der Erkrankung oder die Wirksamkeit und Verträglichkeit von Behandlungen, in den Fokus.…”

Rare disease education in Europe and beyond: time to act

“…Importantly, PLWRD and their advocacy organizations should be included at all stages of the pyramid: they need knowledge and skills for the empowerment, active and meaningful participation in self-care, and advocacy and leadership skills to engage and partner with HWF, researchers, policy makers and regulatory agencies. As PLWRD are experts of their own disease, they could be consulted and engaged into the creation and alignment of educational contents to unmet needs, provision of medical and peer-to-peer education [58][59][60].…”

Psychiatric Disorders and Their Treatment: Impact of Outcomes in Patients With Chronic Liver Disease

“…In less prevalent liver diseases, including immune‐related, cholestatic, and genetic CLDs, studies assessing the impact of behavioral health treatment on CLD outcomes are lacking. In primary sclerosing cholangitis, however, it has been shown that improved self‐management through a structured intervention improved HRQOL, coping, and care navigation 21 . In Wilson’s disease, it is known that decoppering does not reduce the burden of psychiatric disease; thus, lifelong management of comorbid psychiatric disorders is likely needed as part of comprehensive care 22 …”

“…In primary sclerosing cholangitis, however, it has been shown that improved self-management through a structured intervention improved HRQOL, coping, and care navigation. 21 In Wilson's disease, it is known that decoppering does not reduce the burden of psychiatric disease; thus, lifelong management of comorbid psychiatric disorders is likely needed as part of comprehensive care.…”