Key Information
Background
Amyloidosis represents a rare yet heterogeneous multi-system disorder associated with a grave prognosis and an enormous psycho-emotional strain on patients, relatives, and caregivers. We here present the overall study design and first results of AMY-NEEDS, a research program aiming to systematically assess the needs of patients suffering from amyloidosis, their relatives and health care professionals (HCPs), and develop an amyloidosis-specific care approach.
Methods
AMY-NEEDS uses a mixed-methods approach including focus groups (step 1), a questionnaire-based broad evaluation within the local amyloidosis patient collective (step 2), and the development of a needs-adapted care concept (step 3).
Results
Seven patients, six relatives and five HCPs participated in the focus groups (step 1). At the time of diagnosis, patients expressed the need of a smooth diagnostic process, possibly enhanced through improved awareness and better education of local HCPs. There was a strong wish to receive well-founded information and comprehensive support including companionship during medical visits, experience the feeling of being understood, find trust in that “everything possible” is being done, and have effortless access to centre staff. In the course of the disease, patients favoured that the specialized centre should manage treatment coordination, monitoring and psychosocial support. The interface between centre and local HCPs was regarded of particular importance, requiring further investigation into its optimal design.
Conclusions
Patients with amyloidosis express particular needs that should appropriately be considered in specifically tailored care concepts.
Supplementary Information
The online version contains supplementary material available at 10.1186/s13023-024-03052-w.
Introduction & background
Systemic amyloidosis represents a rare, multi-system disorder resulting from deposition of misfolded proteins in the tissue and consecutive organ dysfunction [1external link, opens in a new tab]. The diagnosis of amyloidosis is frequently delayed, because the clinical phenotype is very diverse and lacks early specific signs and symptoms [1external link, opens in a new tab–4external link, opens in a new tab]. The complexity of this multi-system disease requires interdisciplinary centre-based structures for appropriate diagnostic and therapeutic measures, and facilitation of the interaction of all affected persons, including relatives as well as local and centre-associated health care professionals (HCPs). The journey to diagnosis is burdened with frustrations and disappointments for affected patients associated with a high degree of psycho-emotional stress before, during, and after diagnosis [3external link, opens in a new tab].
Previous studies mainly focused on quality of life [5external link, opens in a new tab], and patients with systemic light chain (AL) and hereditary transthyretin (ATTR) amyloidosis [5external link, opens in a new tab–7external link, opens in a new tab]. Subtype-spanning studies also considering treatment requiring ICD-10-coded psychological disorders as well as the type and the extent of treatment-relevant needs of patients and their relatives are lacking. More detailed knowledge of the needs concurring along the diagnostic and therapeutic “journey” of affected individuals, their relatives and carers is required to facilitate needs-adopted care programs that improve adherence and reduce distress.
The AMY-NEEDS research and care program aims to systematically assess the needs among affected patients with amyloidosis, their relatives and HCPs, and to develop a tailored care concept. Here, we present the rationale, design, and first results of the AMY-NEEDS program.
Patients & methods
Overall design of AMY-NEEDs& aims
AMY-NEEDS was designed as substudy of the local amyloidosis prospective cohort study “AmyKoS”. AmyKoS represents a non-interventional single-centre study at the Interdisciplinary Amyloidosis Center of Northern Bavaria founded in 2018, which includes all patients with suspected or proven amyloidosis who undergo diagnostics and/or treatment at the centre. It aims to systematically collect clinical data as well as biomaterials. Patients are recruited in the outpatient department or during hospitalization by the treating physicians. The AMY-NEEDS research and care program employs a multi-step mixed-methods approach comprising three consecutive phases (Fig. 1external link, opens in a new tab).