Real-world treatment patterns, resource use and costs of treating uncontrolled carcinoid syndrome and carcinoid heart disease: a retrospective Swedish study

Abstract

Objectives: To quantify healthcare resource use (HRU) and costs in relation to carcinoid syndrome (CS) and carcinoid heart disease (CHD) in a real-world setting, and to provide perspective on treatment patterns. Materials and methods: Patient data and HRU were collected retrospectively from three Swedish healthcare registers. Adult patients diagnosed with metastatic gastroenteropancreatic neuroendocrine tumors (GEP-NETs) grade 1 or 2 and CS who purchased somatostatin analogs (SSAs), and experienced controlled (defined by SSAs use) and uncontrolled (defined by SSAs dose escalation) CS for !8 months during the study period were included. Patients diagnosed with CHD from the date of the GEP-NET diagnosis were included in the CHD study group. Results: Overall, total HRU cost increased with uncontrolled CS and CHD. Total resource cost was 15,500e/patient during controlled CS (8 months), rising to 21,700e/patient during uncontrolled CS (8 months), representing an increase of $40% (6200e/patient). Costs/patient were driven mainly by SSA use, tumor-related medical interventions and examinations. The total mean cost/year of disease was 1100e/patient without CHD, compared to 4600e/patient with CHD, a difference of 3500e/patient. Excluding SSA cost burden, the main drivers of increased cost in CHD patients were surgical interventions and echocardiography. Conclusions: This study provides a comprehensive overview of the treatment patterns and burden of uncontrolled CS symptoms and CHD using Swedish national register data. Increases in medical interventions and examinations HRU and increased SSA use suggest that SSA dose escalation alone may not effectively control the symptoms associated with uncontrolled CS, highlighting an unmet treatment need in this patient group.

Differential diagnosis of diarrhoea in patients with neuroendocrine tumours: A systematic review

“…One study captured by this review reported improvements to patient QoL after undergoing differential diagnosis of diarrhoea, and other studies have shown that a higher number of bowel movements are associated with lower QoL in patients with GEP-NETs[ 7 , 75 ]. Furthermore, a real-world evidence study reported that total healthcare resource use costs increased with uncontrolled CS in patients with GEP-NETs by up to 40% per patient, when compared with controlled CS[ 76 ]. This highlights an important opportunity for improving QoL in patients with GEP-NETs and efficient use of clinical resources; identifying the true cause(s) of diarrhoea may facilitate optimal symptom control through targeted management.…”

A Budget Impact Model of the Addition of Telotristat Ethyl Treatment to the Standard of Care in Patients with Uncontrolled Carcinoid Syndrome

“…To incorporate CaHD into the model, the following assumptions were made: an increase in 5-HIAA levels is associated with an increase in CaHD incidence; TE reduces 5-HIAA levels regardless of durable response status [38]; and individuals with CaHD have an increased risk of death compared with those [38], in which 20 of 45 (44.4%) patients randomised to SSA + TE demonstrated a durable response, compared with 9 of 45 (20%) patients randomised to SSA + placebo; therefore, for the purpose of this model, 24.4% of the response was presumed to be attributable to TE d Values from Lesén et al [33], which cover an 8-month period, were divided by eight to determine per-month costs e Annual CaHD incidence was assumed to be 2.96% for patients with 5-HIAA < 300 mmol/24 h [50] f TE not included in standard of care…”

“…Patients in the SSA + TE arm during the initial treatment period and those in the durable response and inadequate response to TE health states within the Markov model incurred this cost. Real-world CS and CaHD-related costs, including healthcare resource use (frequency per month), drug acquisition and SSA dosage (cost of average dose) for patients with controlled and uncontrolled CS were obtained from a retrospective Swedish database study [33] (Table 1). A number of symptoms not limited to diarrhoea may contribute to cost differences between responders and non-responders in the model.…”

“…Few options exist for patients whose CS symptoms remain uncontrolled despite treatment with SSAs. The limited alternative treatment options include costly, invasive medical interventions that target tumour load and decrease serotonin secretion [30,33].…”

“…The high healthcare cost of CS, particularly for patients with CS that is uncontrolled by the current standard of care, presents a healthcare, societal, and patient burden. A 2018 Swedish study found the cost per patient with controlled CS over an 8-month period to be €15,500, rising to €21,700 per patient with uncontrolled CS [33]. Furthermore, CS symptoms can be debilitating for patients and have a marked impact on health-related quality of life [22,[40][41][42]; diarrhoea and flushing, in particular, have been associated with reduced physical functioning, increased pain, sleep disturbance, depression and anxiety [41].…”