Personalized, interdisciplinary patient pathway for cross-sector care of multimorbid patients (eliPfad trial): study protocol for a randomized controlled trial.

BACKGROUND: Multimorbid and frail elderly patients often carry a high burden of treatment. Hospitalization due to the onset of an acute illness can disrupt the fragile balance, resulting in further readmissions after hospital discharge. Current models of care in Germany do not meet the needs of this patient group. Rather lack of coordination and integration of care combined with a lack of interdisciplinary approaches result in fragmented and inadequate care and increase the burden of treatment even more. METHODS: eliPfad is a randomized controlled trial conducted in 6 hospitals in Germany. Multimorbid elderly patients aged 55 or older are randomly assigned to the intervention or control group. Patients in the intervention group receive the eliPfad intervention additional to standard care. The core components of eliPfad are: Early assessment of patients' individual treatment burden and support through a specially trained case manager Involvement of the patient's general practitioner (GP) right from the beginning of the hospital stay Preparation of an individual, cross-sectoral treatment plan through the interdisciplinary hospital team with the involvement of the patient's GP Establishment of a cross-sectoral electronic patient record (e-ePA) for documentation and cross-sectoral exchange Support/Promote patient adherence Tailored early rehabilitation during the hospital stay, which is continued at home Close-tele-monitoring of medically meaningful vital parameters through the use of tablets, digital devices, and personal contacts in the home environment The intervention period begins in the hospital and continues 6 weeks after discharge. Patients in the control group will be treated according to standard clinical care and discharged according to current discharge management. The primary aim is the prevention/reduction of readmissions in the first 6 months after discharge. In addition, the impact on health-related quality of life, the burden of treatment, survival, self-management, medication prescription, health literacy, patient-centered care, cost-effectiveness, and process evaluation will be examined. Nine hundred forty-eight patients will be randomized 1:1 to intervention and control group. DISCUSSION: If eliPfad leads to fewer readmissions, proves (cost-)effective, and lowers the treatment burden, it should be introduced as a new standard of care in the German healthcare system. TRIAL REGISTRATION: The trial was registered in the German Clinical Trials Registry (Deutsches Register Klinischer Studien (DRKS)) on 08/14/2023 under the ID DRKS00031500 .

Five years of caplacizumab - lessons learned and remaining controversies in immune-mediated thrombotic thrombocytopenic purpura.

Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare hematologic disease caused by autoantibodies against ADAMTS-13 that trigger microangiopathic hemolytic anemia. Therapeutic plasma exchange and glucocorticoids have been the mainstay of treatment for the past 30 years. In 2019, caplacizumab was approved as an addition to this regimen for the acute treatment of iTTP. Randomized controlled trials and real-world evidence have shown that caplacizumab reduces the time to platelet count normalization, refractoriness, and exacerbations of the disease, with an acceptable safety profile. In the past 5 years, there have been arguments against the upfront use of caplacizumab in all patients with iTTP, particularly related to the perceived lack of clinical benefit, safety concerns related to bleeding risk, and high costs. This perspective aimed to address these concerns in the context of the experience of expert centers that have used the drug for >5 years.

Repetitive administration of rituximab can achieve and maintain clinical remission in patients with MCD or FSGS.

Minimal change disease (MCD) and focal segmental glomerulosclerosis (FSGS) are glomerulopathies associated with nephrotic syndrome. Primary forms of these diseases are treated with various regimes of immunosuppression. Frequently relapsing or glucocorticoid-dependent courses remain challenging. Here, a B-cell-depleting strategy with rituximab represents a salvage option although data are sparse in the adult population. In particular, there is limited evidence on the efficacy of restoring remission after initial successful treatment with rituximab and whether patients benefit from an individualized, relapse-based approach. We identified 13 patients who received multiple therapies with rituximab from the FOrMe-registry (NCT03949972), a nationwide registry for MCD and FSGS in Germany, or from the University Hospital of Cologne. Disease status, changes in serum creatinine, proteinuria, and time to relapse were evaluated. Relapse-free survival was compared to the patients' previous therapy regimens. Through all treatment cycles, an improvement of disease activity was shown leading to a complete remission in 72% and partial remission in 26% after 3 ([Formula: see text]0.001) and 6 months ([Formula: see text]0.001). Relapse-free survival increased from 4.5 months (95%-CI 3-10 months) to 21 months (95%-CI 16-32 months) ([Formula: see text]0.001) compared to previous immunosuppression regimens with no loss in estimated glomerular filtration over time (p = 0.53). Compared to continuous B-cell depletion, an individualized relapse-based approach led to a reduced rituximab exposure and significant cost savings. Relapse-based administration of rituximab in patients with MCD/FSGS with an initial good clinical response did not result in a decreased efficacy at a median follow-up duration of 110 months. Thus, reinduction therapies may provide an alternative to continuous B-cell-depletion and reduce the long-term side effects of continuous immunosuppression.

[Acquired thrombotic-thrombocytopenic purpura - patient management with the advent of novel therapeutic agents]. / Erworbene thrombotisch-thrombozytopenische Purpura ­ Patientenmanagement im Licht neuer Therapien.

HISTORY: A 42-year-old female patient presented with pyelonephritis. Two days later she complained of hematomas and petechia of the extremities. DIAGNOSIS/CLINICAL FINDINGS: Acquired thrombotic thrombocytopenic purpura was diagnosed. Laboratory result showed a thrombotic microangiopathy and thrombocytopenia of 8 × 10E9/l. ADAMTS13-activity was reported to be reduced and inhibitory antibodies to be present. THERAPY AND COURSE: The patient was placed on a therapy with plasma exchange, high-dose steroids, and caplacizumab based on a positive PLASMIC score. Once the diagnosis was confirmed by diminished ADAMTS13-activity and the presence of autoantibodies, rituximab was added. The patient responded well but showed persistently elevated levels of ADAMTS13-autoantibodies, which lead to an extension of Caplacizumab treatment to 58 days. DISCUSSION: Caplacizumab represents a novel effective treatment option for patients with acquired thrombocytopenic purpura. Time to treatment response as well as the risk of relapse were shown to be significantly reduced. However, persistent autoimmune activity can be demonstrated in a significant portion of the patients after the end of treatment putting them at risk for relapses. Thus, careful and frequent surveillance is required.