TY - JOUR
T1 - Biochemical control during long-Term follow-up of 230 adult patients with cushing disease
T2 - A multicenter retrospective study
AU - Geer, Eliza B.
AU - Shafiq, Ismat
AU - Murray, B. Gordon
AU - Bonert, Vivien
AU - Ayala, Alejandro
AU - Swerdloff, Ronald S.
AU - Katznelson, Laurence
AU - Lalazar, Yelena
AU - Manuylova, Ekaterina
AU - Pulaski-Liebert, Karen J.
AU - Carmichael, John D.
AU - Hannoush, Zeina
AU - Surampudi, Vijaya
AU - Broder, Michael S.
AU - Cherepanov, Dasha
AU - Eagan, Marianne
AU - Lee, Jackie
AU - Said, Qayyim
AU - Neary, Maureen P.
AU - Biller, Beverly M.K.
N1 - Funding Information:
This study was funded by Novartis Pharmaceuticals Corporation.
Funding Information:
M.P.N., Q.S., and Y.L. are employees of the Novartis Pharmaceuticals Corporation. Y.L. was affiliated with the Icahn School of Medicine at Mount Sinai throughout the study duration. M.S.B., D.C., M.E., and J.L. are employees of the Partnership for Health Analytic Research, LLC, a health services research company paid by Novartis to conduct this research. J.C. has been Principal Investigator (PI) of research grants to the University of Southern California from Novartis, Pfizer, and Cortendo, has received research support from Novartis, and has performed consulting for Novartis and Pfizer. B.M.K.B. has been the PI of research grants to Massachusetts General Hospital from Cortendo and Novartis and has performed occasional consulting for Cortendo, Ipsen, and Novartis. E.B.G. was the PI of a research grant to Mount Sinai Hospital from Novartis, is PI for clinical trials funded by Novartis and Strongbridge, and has performed occasional consulting for Strongbridge, Chiasma, and Novartis. A.A. has been PI of research grants to the University of Miami from Novartis and has performed consulting for Novartis, Pfizer, and NHT therapeutics. E.M. and I.S. have received honorarium from Novartis. K.P.L., V.B., R.S.S., and V.S. have no conflicts of interest to disclose.
PY - 2017/8
Y1 - 2017/8
N2 - Objective: Cushing disease (CD) results from excessive exposure to glucocorticoids caused by an adrenocorticotropic hormone-secreting pituitary tumor. Inadequately treated CD is associated with significant morbidity and elevated mortality. Multicenter data on CD patients treated in routine clinical practice are needed to assess treatment outcomes in this rare disorder. The study purpose was to describe the burden of illness and treatment outcomes for CD patients. Methods: Eight pituitary centers in four U.S. regions participated in this multicenter retrospective chart review study. Subjects were CD patients diagnosed at ≥18 years of age within the past 20 years. Descriptive statistical analyses were conducted to examine presenting signs, symptoms, comorbidities, and treatment outcomes. Results: Of 230 patients, 79% were female (median age at diagnosis, 39 years; range, 18 to 78 years). Length of follow-up was 0 to 27.5 years (median, 1.9 years). Pituitary adenomas were 0 to 51 mm. The most common presenting comorbidities included hypertension (67.3%), polycystic ovary syndrome (43.5%), and hyperlipidemia (41.5%). Biochemical control was achieved with initial pituitary surgery in 41.4% patients (91 of 220), not achieved in 50.0% of patients (110 of 220), and undetermined in 8.6% of patients (19 of 220). At the end of follow-up, control had been achieved with a variety of treatment methods in 49.1% of patients (110 of 224), not achieved in 29.9% of patients (67 of 224), and undetermined in 21.0% of patients (47 of 224). Conclusion: Despite multiple treatments, at the end of follow-up, biochemical control was still not achieved in up to 30% of patients. These multicenter data demonstrate that in routine clinical practice, initial and long-Term control is not achieved in a substantial number of patients with CD.
AB - Objective: Cushing disease (CD) results from excessive exposure to glucocorticoids caused by an adrenocorticotropic hormone-secreting pituitary tumor. Inadequately treated CD is associated with significant morbidity and elevated mortality. Multicenter data on CD patients treated in routine clinical practice are needed to assess treatment outcomes in this rare disorder. The study purpose was to describe the burden of illness and treatment outcomes for CD patients. Methods: Eight pituitary centers in four U.S. regions participated in this multicenter retrospective chart review study. Subjects were CD patients diagnosed at ≥18 years of age within the past 20 years. Descriptive statistical analyses were conducted to examine presenting signs, symptoms, comorbidities, and treatment outcomes. Results: Of 230 patients, 79% were female (median age at diagnosis, 39 years; range, 18 to 78 years). Length of follow-up was 0 to 27.5 years (median, 1.9 years). Pituitary adenomas were 0 to 51 mm. The most common presenting comorbidities included hypertension (67.3%), polycystic ovary syndrome (43.5%), and hyperlipidemia (41.5%). Biochemical control was achieved with initial pituitary surgery in 41.4% patients (91 of 220), not achieved in 50.0% of patients (110 of 220), and undetermined in 8.6% of patients (19 of 220). At the end of follow-up, control had been achieved with a variety of treatment methods in 49.1% of patients (110 of 224), not achieved in 29.9% of patients (67 of 224), and undetermined in 21.0% of patients (47 of 224). Conclusion: Despite multiple treatments, at the end of follow-up, biochemical control was still not achieved in up to 30% of patients. These multicenter data demonstrate that in routine clinical practice, initial and long-Term control is not achieved in a substantial number of patients with CD.
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U2 - 10.4158/EP171787.OR
DO - 10.4158/EP171787.OR
M3 - Article
C2 - 28614003
AN - SCOPUS:85029788866
VL - 23
SP - 962
EP - 970
JO - Endocrine Practice
JF - Endocrine Practice
SN - 1530-891X
IS - 8
ER -