LanroNET — nieinterwencyjne badanie prospektywne oceniające wykorzystanie zasobów medycznych i koszty Lanreotide Autogel 120 mg w populacji polskich pacjentów z objawowymi guzami neuroendokrynnymi

Wstęp: Celem badania LanroNET była ocena wykorzystania zasobów medycznych oraz kosztów objawowego leczenia polskich chorych na nowotwory neuroendokrynne z zastosowaniem lanreotydu autogel 120 mg. Materiał i metody: LanroNET to wieloośrodkowe, nieinterwencyjne, obserwacyjne, prospektywne badanie przeprowadzone w 12 ośrod­kach w Polsce. W badaniu uczestniczyli dorośli chorzy na wydzielające nowotwory neuroendokrynne leczeni lanreotydem autogel 120 mg od przynajmniej 3 miesięcy przed włączeniem do badania. Podczas 24-miesięcznej obserwacji rzeczywistej praktyki klinicznej zbierano dane dotyczące wykorzystania zasobów medycznych oraz przebiegu terapii chorych z wydzielającymi nowotworami neuroendokrynnymi. Wyniki: W badaniu uczestniczyło 54 chorych na wydzielające nowotwory neuroendokrynne. Przeciętny czas stosowania lanreotydu wynosił 1,7 roku (zakres 0,0–2,2 lata). Badanie ukończyło 33 pacjentów, najczęstszą przyczyną przedwczesnego zakończenia leczenia (8/16) była progresja choroby. Całkowity średni koszt wykorzystanych zasobów bez kosztów farmakoterapii oszacowano na 26 307 zł/EUR 6.030,35 na pacjenta/rok. W czasie badania średni odstęp między wstrzyknięciami lanreotydu wynosił 31,7 dni (6,7). Pod koniec obserwacji, po 24 miesiącach od follow-up, 7 pacjentów stosowało 42-dniowe odstępy między dawkami. Średni rzeczywisty koszt lanreotydu autogel 120 mg wyniósł 4216,30 zł/966,49 EUR na pacjenta/28 dni we wspólnej perspektywie płatnika i pacjenta i był niższy o 554,16 zł/127,02 EUR niż koszt stosowania standardowych 28-dniowych odstępów między dawkami. Wnioski: Badanie LanroNET jest pierwszym w Polsce obserwacyjnym dwuletnim badaniem chorych na czynne hormonalnie nowotwo­ry neuroendokrynne żołądkowo-jelitowo-trzustkowe oceniającym koszty codziennej praktyki klinicznej i koszty leczenia lanreotydem autogel.Introduction: The primary objective of the LanroNET study was to evaluate the resource utilisation and cost of symptomatic treatment of patients with neuroendocrine tumours (NET) using lanreotide autogel 120 mg. Material and methods: LanroNET was a multicentre, non-interventional, prospective study conducted at 12 clinical centres in Poland. Eligible patients were adults with symptomatic NET treated with lanreotide autogel 120 mg at least three months before enrolment. During 24-months of observation of real clinical practice, data on medical resource utilisation and the therapy course of patients with symptomatic NET were collected. Results: Fifty-four patients with symptomatic NET were enrolled. The median time of lanreotide exposure was 1.7 years (range: 0.0–2.2). Thirty-three patients completed the study; the most frequent known cause of discontinuation (8/16) was disease progression. The mean cost of consumed resources without the cost of pharmacotherapy was estimated at PLN 26,307/EUR 6030.35 per year. During the study, the mean (SD) interval between injections was 31.7 days (6.7). At the end of observation — after 24 months of follow-up, seven patients were on a 42-day regimen. The average real-world cost of lanreotide autogel 120 mg was PLN 4216.30/EUR 966.49 per patient/28 days from the public payer and patient perspective and was lower by PLN 554.14/EUR 127.02 than the cost for the standard 28-day dosing interval. Conclusions: LanroNET is the first two-year observational study of patients with symptomatic NET evaluating the cost of every-day clinical practice and lanreotide autogel treatment in Poland

Circulating tumor cells and tumor biomarkers in functional midgut neuroendocrine tumors

CALM-NET was a phase IV exploratory study in the UK that aimed to evaluate if the presence of circulating tumour cells (CTCs) at baseline predicted symptomatic response in patients with midgut neuroendocrine tumours (NETs) treated with lanreotide autogel (LAN). Adults with functional, well/moderately differentiated (Ki-67 0. Primary endpoint was the clinical value of baseline CTCs to predict symptomatic response (decrease in diarrhoea or flushing of ≥50% frequency, or ≥1 severity level). Other endpoints included progression-free survival (PFS) and correlations between plasma and urinary biomarkers (including 5-hydroxyindoleacetic acid [5-HIAA]). Fifty patients were enrolled; 40 completed the study. Baseline CTCs were present in 22 (45.8%) patients (missing baseline CTC status n = 2). Overall, 87.5% (95% confidence interval [CI]: 73.9; 94.5) of patients had a symptomatic response; a 5.9-fold higher odds of symptomatic response in patients without CTC versus patients with CTC at baseline was observed, although this was not statistically significant (odds ratio: 0.17 [95% CI: 0.02; 1.65], p = .126). One-year PFS rate was 66.4% (95% CI: 48.8; 79.2). Biomarker concentrations did not correlate to baseline CTC status. However, there was a strong correlation between plasma and urinary 5-HIAA (Spearman correlation coefficients ≥0.87 [p < .001], all time points). In conclusion, patients without CTC at baseline may be more likely to achieve a symptomatic response following LAN treatment than patients with CTC. Plasma 5-HIAA correlated with urinary 5-HIAA during LAN treatment. ClinicalTrials.gov identifier: NCT02075606

Quality of life in patients with acromegaly receiving lanreotide autogel: a real-world observational study

Introduction: Patients with acromegaly have substantially reduced quality of life (QoL). This study evaluated QoL in patients with acromegaly treated with lanreotide autogel. Material and methods: This was a prospective, non-interventional, observational, multi-centre study conducted in Poland (NCT02396966). We included patients with acromegaly, who received treatment with lanreotide autogel 120 mg for ≥ 3 months and &lt; 3 years. Patients were assessed approximately every 4–5 months for two years (six visits). QoL was measured with the Acromegaly Quality of Life Questionnaire (AcroQoL). Results: Of 152 patients enrolled from November 2014 to May 2018 in 37 centres, 24 were excluded due to major protocol deviations. The results are reported for the study population (n = 128). At baseline, the median [95% confidence interval (CI)] time from diagnosis was 3.3 (2.8, 4.2) years, and the median time since lanreotide initiation was 13.4 (9.9, 17.3) months. Symptoms of acromegaly were present at baseline in 86% of patients (headache, 57%; sweating, 58%; joint symptoms, 64%); symptoms remained unchanged at two years in 82% of patients. At baseline, 27% of patients had hormonal control (growth hormone &lt; 2.5 μg/L and insulin-like growth factor-1 within the normal range); hormonal control status did not change during the study period in over 81% of patients. At baseline, 88% of patients were either very satisfied or satisfied with treatment; treatment satisfaction was unchanged in 62% of patients over the study period. Mean (95% CI) AcroQoL scores at baseline were as follows: total, 50.3 (47.3, 53.3); physical dimension, 48.8 (45.2, 52.4); psychological dimension, 51.3 (48.2, 54.4); appearance subdimension, 40.7 (37.5, 43.8); and personal relations subdimension, 62.5 (58.8, 66.2). The psychological appearance subscore improved by 3.8 points (1.2, 6.5) over the two years; scores in the remaining dimensions and subdimensions did not change substantially. The total AcroQoL score remained unchanged over the two years, regardless of prior acromegaly treatment, surgery or radiotherapy, hormonal control, or lanreotide dosing interval. No new safety findings were identified. Conclusions: AcroQoL total scores and physical and psychological subscores remained stable but impaired among patients with long-lasting acromegaly treated with lanreotide autogel for two years. The psychological appearance subdimension improved numerically

Satisfaction with injection experience of patients with neuroendocrine tumors enrolled on lanreotide autogel patient support programs: Results from the international HomeLAN survey

Lanreotide autogel/depot (LAN) is a somatostatin analog used in first-line treatment for neuroendocrine tumors (NETs). The aim of HomeLAN was to evaluate the satisfaction with injection experience among patients with NETs receiving at-home LAN injection via patient support programs (PSPs). This was an international, non-interventional, cross-sectional, online survey in adults with NETs enrolled in PSPs, receiving LAN injections at home, administered by a healthcare professional (HCP) or administered independently (self or caregiver administering injection). The primary endpoint was satisfaction with the most recent LAN injection. Secondary endpoints included the level of anxiety prior to injection, impact on daily life, and the extents to which participants felt in control of their life and agreed that home administration met their medical needs. In total, 111 participants from Belgium, Greece, the Netherlands, and Spain completed the survey (50.5% male; mean age, 63.6 years; most common primary tumor site was intestine [47.7%]). For 99 participants, their most recent injection was administered by an HCP. Overall, 95.5% of all participants were satisfied with their most recent injection experience (95% confidence interval: 89.89%–98.06%); 67% reported experiencing no anxiety prior to injection, 91.0% reported that home injection had a “great deal” or “quite a bit” of positive impact on their daily life, and 85.6% strongly agreed that the PSP met their medical needs. In the HCP injection subgroup, 71.7% reported that this mode of administration helped them to feel in control of their lives. In this patient survey, satisfaction levels were high among patients with NETs receiving LAN injections at home via a LAN PSP. Most patients did not experience anxiety prior to their most recent injection and acknowledged that thanks to their treatment they had a good quality of life despite their disease. Most strongly agreed that the PSP met their medical needs, which highlights the valuable service that LAN PSPs provide for patients with NETs

Treatment Patterns, Adherence, Persistence, and Healthcare Resource Utilization in Acromegaly: A Real-World Analysis

International audienceTreatment of acromegaly is multimodal for many patients, and medical treatments include somatostatin receptor ligands (SRLs), dopamine agonists (DAs), and growth hormone receptor antagonists (GHRAs). However, recent real-world evidence on treatment patterns for patients with acromegaly is limited. This study evaluated medication usage, treatment changes, adherence, persistence, comorbidities, and healthcare resource utilization, using de-identified data from MarketScan®, a US claims database. Eligible patients (n = 882) were those receiving monotherapy or combination therapy for ≥90 days without treatment gaps. Mean age at diagnosis was 48.6 years; 50.1% of patients were female. Over half (59.4%) had one line of treatment (LOT); 23.1% had two LOTs; 17.5% had at least three LOTs. Most patients (94.6%) initiated treatment with monotherapies. The most common first-line monotherapy treatments were cabergoline (DA, 36.8%), octreotide long-acting release (first-generation SRL, 29.5%), and lanreotide depot (first-generation SRL, 22.5%). Adherence for first-line treatments (proportion of days covered), was higher for first-generation SRLs (lanreotide depot: 0.8), compared with DAs (0.7). Treatment persistence (time between the first treatment record and a change in LOT/censoring) in LOT 1 was higher for GHRAs (24.8 months) and first-generation SRLs (20.0 months), compared with DAs (14.4 months). Female patients and those diagnosed at a younger age were more likely to have shorter treatment persistence. The most prevalent comorbidities were hyperlipidemia, essential hypertension, and sleep apnea. Patients with more comorbidities had more healthcare visits during the first year after diagnosis, suggesting increased disease burden. Real-world evidence on treatment patterns provides insights into recommendations for individualized therapy

Evaluating home injection compared with healthcare-setting injection of somatostatin analogs: a systematic literature review

Purpose: A systematic literature review was conducted to assess the use of home injections (self/partner/healthcare provider [HCP]-administered) of somatostatin analogs (SSAs) as an alternative to healthcare-setting injections in patients with acromegaly and neuroendocrine tumors (NETs). Methods: MEDLINE/Embase/the Cochrane Library (2001–September 2021), key congresses (2019–2021), and bibliographies of relevant systematic reviews were searched. Eligible studies reported on efficacy/effectiveness, safety, adherence, patient-reported outcomes (PROs), and economic outcomes in populations receiving home injections of SSAs. Results: Overall, 12 studies were included, all reporting on SSAs (lanreotide Autogel/Depot or octreotide long-acting release) in acromegaly or NETs. Across four studies, home injection was associated with similar disease control in patients with acromegaly/NETs compared with healthcare-setting administration. High rates of treatment adherence were shown in two studies of patients with acromegaly receiving lanreotide injections at home. Two studies reported non-serious adverse events; incidence of adverse reactions was similar in both the home and healthcare administration settings. Preference for injection setting varied between studies and indications; nonetheless, higher satisfaction/convenience (>75% patients) was reported for home injections. Self- or partner-injection was associated with economic savings compared with administration in the healthcare setting across five studies. Conclusion: Efficacy/effectiveness, adherence, and safety outcomes of SSAs in the home injection setting were similar to those in the healthcare setting, with high reported satisfaction and convenience. Self/partner injection also resulted in cost savings. These findings provide a basis to understand outcomes related to home injection and encourage healthcare providers to discuss optimal treatment choices with their patients

Circulating tumour cells and tumour biomarkers in functional midgut neuroendocrine tumours.

CALM-NET was a phase IV exploratory study in the UK that aimed to evaluate if the presence of circulating tumour cells (CTCs) at baseline predicted symptomatic response in patients with midgut neuroendocrine tumours (NETs) treated with lanreotide autogel (LAN). Adults with functional, well/moderately differentiated (Ki-67 0. Primary endpoint was the clinical value of baseline CTCs to predict symptomatic response (decrease in diarrhoea or flushing of ≥50% frequency, or ≥1 severity level). Other endpoints included progression-free survival (PFS) and correlations between plasma and urinary biomarkers (including 5-hydroxyindoleacetic acid [5-HIAA]). Fifty patients were enrolled; 40 completed the study. Baseline CTCs were present in 22 (45.8%) patients (missing baseline CTC status n = 2). Overall, 87.5% (95% confidence interval [CI]: 73.9; 94.5) of patients had a symptomatic response; a 5.9-fold higher odds of symptomatic response in patients without CTC versus patients with CTC at baseline was observed, although this was not statistically significant (odds ratio: 0.17 [95% CI: 0.02; 1.65], p = .126). One-year PFS rate was 66.4% (95% CI: 48.8; 79.2). Biomarker concentrations did not correlate to baseline CTC status. However, there was a strong correlation between plasma and urinary 5-HIAA (Spearman correlation coefficients ≥0.87 [p < .001], all time points). In conclusion, patients without CTC at baseline may be more likely to achieve a symptomatic response following LAN treatment than patients with CTC. Plasma 5-HIAA correlated with urinary 5-HIAA during LAN treatment. ClinicalTrials.gov identifier: NCT02075606