Aude Houchard

SAGIT®: clinician-reported outcome instrument for managing acromegaly in clinical practice—development and results from a pilot study

PurposeThe SAGIT instrument is a comprehensive clinician-reported outcome instrument assessing key features of acromegaly: signs and symptoms, associated comorbidities; growth hormone levels; insulin-like growth factor-1 levels; and tumor profile. The SAGIT instrument has been designed to assist endocrinologists managing acromegaly in practice. Here, we report on pre-testing (to assess ease of understanding and acceptability) and a pilot study (to assess relevance, ease of use, and utility in real-life conditions) (NCT02231593).MethodsFor pre-testing, 11 endocrinologists completed the SAGIT instrument using patient medical records and were also interviewed. They subsequently completed a PRAgmatic Content and face validity Test (PRAC-Test©) to report their experiences using SAGIT, and feedback was used to revise the instrument. In the pilot study, nine endocrinologists completed the SAGIT instrument in real-time with patients belonging to three different categories (stable/controlled, active/uncontrolled acromegaly, treatment-naïve), while four completed the instrument based on medical-record review. All participants then completed the PRAC-Test© and their feedback was used to update the instrument.ResultsThe SAGIT instrument was well accepted by endocrinologists, with most indicating that it was concise, practical, easy to understand, useful for assessing treatment response, and valuable as a component of the patient’s medical record. The pilot study confirmed the instrument’s acceptability, utility, and ease of use, and indicated its potential for distinguishing acromegaly clinical stages.ConclusionsThe SAGIT instrument is promising as a tool for use by endocrinologists in everyday practice to assess the status and evolution of disease in patients with acromegaly and to guide treatment decision-making.

Recombinant human growth hormone plus recombinant human insulin-like growth factor-1 coadministration therapy in short children with low insulin-like growth factor-1 and growth hormone sufficiency: Results from a randomized, multicenter, open-label, parallel-group, active treatment-controlled trial

Background/Aims: Growth hormone (GH) and insulin-like growth factor-1 (IGF-1) both contribute to growth. To determine if recombinant human (rh)GH + rhIGF-1 therapy is more effective than rhGH alone to treat short stature, we assessed the efficacy and safety of coadministered rhGH + rhIGF-1 in short children with GH sufficiency and low IGF-1. Methods: In a 3-year, randomized, multicenter, open-label trial, patients with height SD score ≤−2.0 and IGF-1 SD score ≤−1.0 for age and sex, and with stimulated GH ≥10 ng/ml for age and sex, were randomized to receive (all doses in µg/kg/day): 45 rhGH alone (group A), 45 rhGH + 50 rhIGF-1 (group B), 45 rhGH + 100 rhIGF-1 (group C) or 45 rhGH + 150 rhIGF-1 (group D). Height velocity (HV) and Δ height SD score were measured. Results: The first-year HV (modified intention-to-treat population) was 9.3 ± 1.7 cm/year (group A), 10.1 ± 1.3 cm/year (group B), 9.7 ± 2.5 cm/year (group C) and 11.2 ± 2.1 cm/year (group D) (p = 0.001 for groups A vs. D). This effect was sustained, resulting in a height SD score improvement during the second and third years. Most treatment-emergent adverse events were mild and transient. Conclusion: In children with short stature, GH sufficiency and low IGF-1, coadministration of rhGH/rhIGF-1 (45/150 µg/kg) significantly accelerated linear growth compared with rhGH alone, with a safety profile similar to the individual monotherapies.

Factors predicting progression to castrate-resistant prostate cancer in patients with advanced prostate cancer receiving long-term androgen-deprivation therapy

To assess time to progression to castrate‐resistant prostate cancer (CRPC) and factors influencing longer‐term outcomes in patients receiving androgen‐deprivation therapy (ADT) in an extension to the Triptocare study (NCT01020448). This is pertinent as the Triptocare study did not show that urinary prostate cancer antigen‐3 (PCA3) score was a reliable marker of cancer stage in advanced prostate cancer and was not useful for assessing response 6 months after initiation of ADT with triptorelin 22.5 mg.

Glucose and lipid levels with lanreotide autogel 120 mg in treatment-naïve patients with acromegaly: data from the PRIMARYS study.

Impaired glycaemic control, characteristic of acromegaly, can be exacerbated by treatment with somatostatin analogues (SSAs), particularly those with multireceptor activity. We present data from the PRIMARYS study on the impact of the SSA lanreotide, associated with tumour volume and hormonal improvements, on glucose and other metabolic parameters in acromegaly.

LanroNET — A Non-Interventional Prospective Study to Assess the Resource Utilisation and Cost of Lanreotide Autogel 120 mg in the Population of Polish Patients with Symptomatic Neuroendocrine Tumours

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 ośrod-kach 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 nowotwo-ry neuroendokrynne żołądkowo-jelitowo-trzustkowe oceniającym koszty codziennej praktyki klinicznej i koszty leczenia lanreotydem autogel.

Signs and symptoms of acromegaly at diagnosis: the physician’s and the patient’s perspectives in the ACRO-POLIS study

PurposeAcromegaly is characterized by a broad range of manifestations. Early diagnosis is key to treatment success, but is often delayed as symptomatology overlaps with common disorders. We investigated sign-and-symptom associations, demographics, and clinical characteristics at acromegaly diagnosis.MethodsObservational, cross-sectional, multicenter non-interventional study conducted at 25 hospital departments in France that treat acromegaly (ClinicalTrials.gov: NCT02012127). Adults diagnosed with acromegaly < 5 years were enrolled. Demographic and clinical data were obtained from medical reports and patient questionnaires. Sign-and-symptom associations were assessed by multiple correspondence analysis (MCA).ResultsOverall, 472 patients were included in the analyses. MCA was unsuccessful in identifying sign-and-symptom associations at diagnosis. Endocrinologists (29.5% patients) and other clinical specialists (37.2% patients) were commonly first to suspect acromegaly. Morphologic manifestations (83.7–87.9% patients), snoring syndrome (81.4% patients), and asthenia (79.2% patients) were frequently present at diagnosis; differences were found between sexes for specific manifestations. Rates of discrepancy between patient- and physician-reported manifestations were highest for functional signs. Earliest manifestations prior to diagnosis, according to how they were detected, were enlarged hands and feet (6.4 ± 6.8 and 6.2 ± 6.9 years, functional signs), hypertension (6.6 ± 7.5 years, complementary examination) and carpal/cubital tunnel syndrome (5.7 ± 6.7 years, functional signs with complementary examination).ConclusionsResults confirm the broad range of manifestations at diagnosis and delay in recognizing the disease. We identified early manifestations and sex differences that may aid physicians in diagnosing acromegaly. Discrepancy rates suggest physicians should obtain the patient’s perspective and seek functional signs during diagnosis.