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Long-term treatment outcomes of acromegaly patients presenting biochemically-uncontrolled at a tertiary pituitary center.

ABSTRACT: Acromegaly is a rare, slowly progressive disorder resulting from excessive growth hormone (GH) production by a pituitary somatotroph tumor. The objective of this study was to examine acromegaly treatment outcomes during long-term care at a specialized pituitary center in patients presenting with lack of biochemical control.Data came from an acromegaly registry at the Cedars-Sinai Medical Center Pituitary Center (center). Acromegaly patients included in this study were those who presented biochemically-uncontrolled for care at the center. Biochemical control status, based on serum insulin-like growth factor-1 values, was determined at presentation and at study end. Patient characteristics and acromegaly treatments were reported before and after presentation by presenting treatment status and final biochemical control status. Data on long-term follow-up were recorded from 1985 through June 2013.Seventy-four patients presented uncontrolled: 40 untreated (54.1%) and 34 (45.9%) previously-treated. Mean (SD) age at diagnosis was 43.2 (14.7); 32 (43.2%) were female patients. Of 65 patients with tumor size information, 59 (90.8%) had macroadenomas. Prior treatments among the 34 previously-treated patients were pituitary surgery alone (47.1%), surgery and medication (41.2%), and medication alone (11.8%). Of the 40 patients without prior treatment, 82.5% achieved control by study end. Of the 34 with prior treatment, 50% achieved control by study end.This observational study shows that treatment outcomes of biochemically-uncontrolled acromegaly patients improve with directed care, particularly for those that initially present untreated. Patients often require multiple modalities of treatment, many of which are offered with the highest quality at specialized pituitary centers. Despite specialized care, some patients were not able to achieve biochemical control with methods of treatment that were available at the time of their treatment, showing the need for additional treatment options.

SUBMITTER: Carmichael JD

PROVIDER: S-EPMC5545017 | biostudies-other | 2017 Aug

REPOSITORIES: biostudies-other

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Project description:Unlike other complications among very low birth weight infants (VLBW), the incidence of bronchopulmonary dysplasia (BPD) has not decreased substantially, partly because of the different definitions of BPD applied by different researchers. In this retrospective cohort study, we aimed to compare the 2018 revised definition and the 2001 consensus definition of BPD proposed by the National Institute of Child Health and Human Development (NICHD), as well as to identify which definition better predicts severe respiratory morbidities or death. We included 417 infants born at a gestational age <32 weeks and classified them as having BPD or without BPD based on the two definitions, with a final follow-up at 18-24 months. We performed between-group comparisons of death and respiratory outcomes. Statistical analyses were performed using descriptive statistics, comparative tests, and receiver operating characteristic curves. The mean ± standard deviation gestational age and birth weight of the 417 eligible infants were 29.1 ± 1.4 weeks and 1186.6 ± 197.8 g, respectively. Among the included infants, five and three infants died before and after 36 weeks of post-menstrual age (PMA), respectively, with 68 and 344 infants evaluated at discharge and 36 weeks' PMA, respectively. We diagnosed 163 (39.1%) and 70 (16.8%) infants with BPD according to the 2001 and 2018 NICHD definitions, respectively. The 2001 NICHD definition displayed a higher sensitivity (0.60 vs. 0.28), better negative predictive value (0.89 vs. 0.85), and larger area under the receiver operating characteristic curve (0.66 vs. 0.57), but a lower specificity (0.65 vs. 0.87) and worse positive predictive value (0.26 vs. 0.31), than the 2018 definition for serious respiratory morbidity or mortality at a corrected age of 18-24 months. Compared with the 2018 NICHD definition of BPD, the 2001 NICHD consensus definition may result in more cases of false-positive or unclassified severity. However, it may be a better indicator of severe respiratory morbidities or death during the first 18-24 months. Nevertheless, there is a need for future studies to assess the validity of the new diagnostic criteria.

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Long-term treatment outcomes of acromegaly patients presenting biochemically-uncontrolled at a tertiary pituitary center.

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