Dawkowanie i koszt lanreotydu Autogel 120 mg stosowanego w leczeniu akromegalii w Polsce — wyniki 2-letniego badania Lanro-Study

Introduction: To evaluate, over 24 months of prospective follow-up, the dosage and costs of lanreotide AUTOGEL 120 mg (ATG120) administered as part of routine acromegaly care in Poland.Material and methods: A multicentre, non-interventional, observational prospective study on resource utilisation in Polish acromegalic patients treated with ATG120 at 4-week or extended (> 4 weeks) dosing interval. The study population consisted of adult acromegalic patients treated for at least 3 injections of ATG120. The endpoints were: percentage of patients treated with ATG120 at an extended dosing interval (> 4 weeks), mean time between injections, and the cost of ATG120 during a 24-month prospective observation. Costs were calculated in PLN from the public health-care payer and patient perspective for the year 2014.Results: 143 patients were enrolled in, and 132 completed (70% women, 81% macroadenoma, 75% previous surgery) the analysis. During two years, changes in the treatment pattern were reported in 41 patients: 17% of them had increased injection interval and 10% switched to octreotide LAR and then returned to ATG120. Sixty-three patients (48%) received ATG120 at an extended dosing interval. ATG120 was predominantly administered in an out-patient setting (84%) by a health care professional (97%).Conclusions: The results demonstrated that extended dosing interval of ATG120 is used in a substantial proportion of patients in routine clinical practice in Poland. Such findings support the potential for ATG120 in reducing treatment burden in the real-world clinical environment. (Endokrynol Pol 2015; 66 (2): 142–148)Wstęp: Ocena schematu dawkowania i kosztu lanreotydu Autogel 120 mg (ATG120) stosowanego w leczeniu akromegalii w Polsce.Materiał i metody: Wieloośrodkowe, nieinterwencyjne, obserwacyjne badanie prospektywne oceniające zużycie zasobów ochrony zdrowia w populacji polskich pacjentów z akromegalią leczonych ATG120 z zastosowaniem 4-tygodniowego lub wydłużonego (> 4 tygodni) odstępu między dawkami leku. Do badania włączani byli dorośli pacjenci, którzy otrzymali co najmniej 3 dawki ATG120. Horyzont czasowy badania wynosił 24 miesięcy. Koszt leku obliczono z perspektywy płatnika publicznego i pacjenta w 2014 roku.Wyniki: Do badania włączono 143 pacjentów, 132 z nich ukończyło badanie (70% kobiet, u 81% makrogruczolak, 75% było wcześniej leczonych chirurgicznie). W czasie 2 lat zmiany schematu leczenia dokonano u 41 chorych, u 17% z nich wydłużono odstęp między dawkami, u 10% — zmieniono lek na oktreotyd LAR a potem ponownie zastosowano ATG120. U 63 pacjentów (48%) odstęp pomiędzy dawkami ATG120 wynosił > 4 tygodnie. ATG120 był przeważnie podawany ambulatoryjnie (84%) przez fachowy personel medyczny (97%). Wnioski: Badanie wykazało, że w codziennej praktyce klinicznej u znaczącego odsetka pacjentów odstęp czasowy pomiędzy kolejnymi dawkami ATG120 jest dłuższy niż 4 tygodnie. Wynik ten potwierdza tezę, że dzięki stosowaniu ATG120 można obniżyć koszty leczenia akromegalii. (Endokrynol Pol 2015; 66 (2): 142–148

Pulmonary neuroendocrine (carcinoid) tumors: European Neuroendocrine Tumor Society expert consensus and recommendations for best practice for typical and atypical pulmonary carcinoids

This is an expert consensus from the European Neuroendocrine Tumor Society recommending best practice for the management of pulmonary neuroendocrine tumors including typical and atypical carcinoids. It emphasizes the latest discussion on nomenclature, advances and utility of new diagnostic techniques as well as the limited evidence and difficulties in determining the optimal therapeutic strateg

The clinical applications of a multigene liquid biopsy (NETest) in neuroendocrine tumors

Purpose: There are few effective biomarkers for neuroendocrine tumors. Precision oncology strategies have provided liquid biopsies for real-time and tailored decision-making. This has led to the development of the first neuroendocrine tumor liquid biopsy (the NETest). The NETest represents a transcriptomic signature of neuroendocrine tumor (NETs) that captures tumor biology and disease activity. The data have direct clinical application in terms of identifying residual disease, disease progress and the efficacy of treatment. In this overview we assess the available published information on the metrics and clinical efficacy of the NETest. Material and methods: Published data on the NETest have been collated and analyzed to understand the clinical application of this multianalyte biomarker in NETs. Results: NETest assay has been validated as a standardized and reproducible clinical laboratory measurement. It is not affected by demographic characteristics, or acid suppressive medication. Clinical utility of the NETest has been documented in gastroenteropancreatic, bronchopulmonary NETs, in paragangliomas and pheochromocytomas. The test facilitates accurate diagnosis of a NET disease, and real-time monitoring of the disease status (stable/progressive disease). It predicts aggressive tumor behavior, identifies operative tumor resection, and efficacy of the medical treatment (e.g. somatostatin analogues), or peptide receptor radionuclide therapy (PRRT). NETest metrics and clinical applications out-perform standard biomarkers like chromogranin A. Conclusions: The NETest exhibits clinically competent metrics as an effective biomarker for neuroendocrine tumors. Measurement of NET transcripts in blood is a significant advance in neuroendocrine tumor management and demonstrates that blood provides a viable source to identify and monitor tumor status

NETest liquid biopsy is diagnostic of small intestine and pancreatic neuroendocrine tumors and correlates with imaging

Introduction: Current monoanalyte biomarkers are ineffective in gastroenteropancreatic neuroendocrine tumors (GEP-NETs). NETest, a novel multianalyte signature, provides molecular information relevant to disease biology. Aim(s): Independently validate NETest to diagnose GEP-NETs and identify progression in a tertiary referral center. Materials and methods: Cohorts are 67 pancreatic NETs (PNETs), 44 small intestine NETs (SINETs) and 63 controls. Well-differentiated (WD) PNETs, n = 62, SINETs, all (n = 44). Disease extent assessment at blood draw: anatomical (n = 110) CT (n = 106), MRI (n = 7) and/or functional Ga-68-SSA-PET/CT (n = 69) or F-18-FDG-PET/CT (n = 8). Image-positive disease (IPD) was defined as either CT/MRI or Ga-68-SSA-PET/CT/F-18-FDG-PET/CT-positive. Both CT/MRI and Ga-68-SSA-PET/CT negative diagnosis in WD-NETs was considered image-negative disease (IND). NETest (normal: 20): PCR (spotted plate s). Data: mean +/- SD. Results: Diagnosis: NETest was significantly increased in NETs (n = 111; 26 +/- 21) vs controls (8 +/- 4, p < 0.0001). Seventy-five (42 PNET, 33 SINET) were image positive. Eleven (8 PNET, 3 SINET; all WD) were IND. In IPD, NETest was significantly high er (36 +/- 22) vs IND (8 +/- 7, P < 0.0001). NETest accuracy, sensitivity and specificity are 97, 99 and 95%, respectively. Concordance with imaging: NETest was 92% (101/110) concordant with anatomical imaging, 94% (65/69) with Ga-68-SSA-PET/CT and 96% (65/68) dual modality (CT/MRI and Ga-68-SSA-PET/CT). In 70 CT/MRI positive, NETest was elevated in all (37 +/- 22). In 40 CT/MRI negative, NETest was normal (11 +/- 10) in 31. In 56 Ga-68-SSA-PET/CT positive, NETest was elevated (36 +/- 22) in 55. In 13 Ga-68-SSA-PET/CT negative, NETest was normal (9 +/- 8) in ten. Disease status: NETest was significantly higher in progressive (61 +/- 26; n = 11) vs stable disease (29 +/- 14; n = 64; P < 0.0001) (RECIST 1.1). Conclusion: NETest is an effective diagnostic for PNETs and SINETs. Elevated NETest is as effective as imaging in diagnosis and accurately identifies progression

The European Neuroendocrine Tumour Society registry, a tool to assess the prognosis of neuroendocrine neoplasms.

Neuroendocrine neoplasms (NENs) are rare tumours with variable clinical behaviour. Their natural history is ideally best approached in large, multicentre and multinational registries with long-term patients' follow-up. The European Neuroendocrine Tumour Society registry aims to obtain information regarding NEN outcomes and prognostic factors in a European frame. We collected data from 7 national NEN registries (Belgium, Czech Republic, Germany, Greece, Poland, Spain, Switzerland), representing 10,102 patients. Anonymised/pseudonymised data were collected in a secured server. Descriptive statistical methods were applied, as well as Kaplan-Meier survival curves and multivariable analyses for prognostic factors of overall survival (OS). median age of the study population was 60 years (range: 18-102), 48% were female. Common primary tumour sites were pancreas (27%) and small intestine (21%). Stage 4 disease was found in 47% of patients, while 26/10/16% had stage 1/2/3 disease, respectively. Grading (n = 6952) was G1/2/3 in 48/37/15% of the patients, respectively. Surgery was the main treatment, provided to 71% of patients, followed by somatostatin analogues (32%), chemotherapy (20%), Peptide receptor Radionuclide Therapy (PRRT) (9%) and targeted therapies (8%). OS at 5 years was 74%, influenced by grade, stage and tissue of origin in multivariate analysis. A Ki67 cut-off value set at 55% within the G3 group allowed to separate 2 groups with a meaningful different OS. We report the first analysis of the European Neuroendocrine Tumour Society registry, comprising 10,102 patients with NEN from 7 European countries. This large cohort study describes prognostic factors for the survival of NENs throughout Europe, including primary tumour site, grade, stage and treatment

ENETS Consensus Guidelines for the Standards of Care in Neuroendocrine Tumors : Pre- and Perioperative Therapy in Patients with Neuroendocrine Tumors

Neuroendocrine tumors of the small intestine are the most common causes of the carcinoid syndrome. Carcinoid heart disease occurs in more than half of the patients with the carcinoid syndrome. Patients with carcinoid heart disease who need to undergo surgery should also undergo preoperative evaluation by an expert cardiologist. Treatment with longacting somatostatin analogs aims at controlling the excessive hormonal output and symptoms related to the carcinoid syndrome and at preventing a carcinoid crisis during interventions. Patients with a gastrinoma require pre- and postoperative treatment with high doses of proton pump inhibitors. Patients with a glucagonoma require somatostatin analog treatment and nutritional supplementation. Patients with a VIPoma also require somatostatin analog treatment and intravenous fluid and electrolyte therapy. Insulinoma patients generally require intravenous glucose infusion prior to operation. In patients with localized operable insulinoma, somatostatin analog infusion should only be considered after the effect of this therapy has been electively studied

Well-differentiated gastric tumors/carcinomas.

[No abstract available

Thyroid diseases in pregnancy: guidelines of the Polish Society of Endocrinology [Choroby tarczycy w ciąży: zalecenia postępowania Polskiego Towarzystwa Endokrynologicznego]

Appropriate care of pregnant women with coexisting thyroid dysfunction is still a subject of much controversy. In recent years, there has been a dynamic increase in the number of scientific reports on the diagnosis and treatment of thyroid diseases in women planning pregnancy, pregnant women, and women in the postpartum period. These mainly concern the management of hypothyroidism, autoimmune thyroid diseases, and fertility disorders. Therefore, the Polish Society of Endocrinology deemed it necessary to update the guidelines on principles of diagnostic and therapeutic management in this group of patients, previously published in 2011. The recommendations were prepared by Polish experts according to evidence based medicine principles, if such data were available.