PMID- 31440946
OWN - NLM
STAT- MEDLINE
DCOM- 20200323
LR  - 20200323
IS  - 1573-7403 (Electronic)
IS  - 1386-341X (Print)
IS  - 1386-341X (Linking)
VI  - 22
IP  - 5
DP  - 2019 Oct
TI  - Long-term safety and efficacy of subcutaneous pasireotide in patients with 
      Cushing's disease: interim results from a long-term real-world evidence study.
PG  - 542-551
LID - 10.1007/s11102-019-00984-6 [doi]
AB  - PURPOSE: Clinical trials have demonstrated the favorable efficacy/safety profile 
      of pasireotide in patients with Cushing's disease (CD). We report interim 
      long-term results of an ongoing real-world evidence study of subcutaneous 
      pasireotide in patients with CD. METHODS: Adults with CD receiving pasireotide, 
      initiated before (prior-use) or at study entry (new-use), were monitored 
      for </= 3 years during a multicenter observational study ( 
      http://clinicaltrials.gov identifier NCT02310269). Primary objective was to 
      assess long-term safety of pasireotide alone or with other CD therapies. RESULTS: 
      At the time of this interim analysis, 127 patients had received pasireotide 
      (new-use, n = 31; prior-use, n = 96). Eight patients had completed the 3-year 
      observation period, 53 were ongoing, and 66 had discontinued. Among 31 new-use 
      and 92 prior-use patients with >/= 1 safety assessment, respectively: 24 (77%) and 
      37 (40%) had drug-related adverse events (AEs); 7 (23%) and 10 (11%) had serious 
      drug-related AEs. Most common drug-related AEs were nausea (14%), hyperglycemia 
      (11%) and diarrhea (11%); these were more frequently reported in new users and 
      mostly of mild-to-moderate severity. 14 (45%) new-use and 15 (16%) prior-use 
      patients experienced hyperglycemia-related AEs. Mean urinary free cortisol (mUFC) 
      was within normal range at baseline and months 1, 12 and 24, respectively, in: 
      1/16 (6%), 9/18 (50%), 1/3 (33%) and 0/0 new users; 28/43 (65%), 15/27 (56%), 
      27/33 (82%) and 12/19 (63%) prior users. CONCLUSIONS: Pasireotide is well 
      tolerated and provides sustained reductions in mUFC during real-world treatment 
      of CD. The lower rate of hyperglycemia-related AEs in prior users suggests that 
      hyperglycemia tends not to deteriorate if effectively managed soon after onset. 
      CLINICAL TRIAL REGISTRATION NUMBER: NCT02310269.
FAU - Manetti, Luca
AU  - Manetti L
AUID- ORCID: 0000-0001-9911-5366
AD  - Azienda Ospedaliero-Universitaria Pisana, Dipartimento di Medicina Clinica e 
      Sperimentale, Universita di Pisa, UO Endocrinologia 2 Ospedale Cisanello Via 
      Paradisa 2, 56124, Pisa, Italy. l.manetti@ao-pisa.toscana.it.
FAU - Deutschbein, Timo
AU  - Deutschbein T
AD  - Division of Endocrinology and Diabetes, Department of Internal Medicine I, 
      University Hospital, University of Wurzburg, Wurzburg, Germany.
FAU - Schopohl, Jochen
AU  - Schopohl J
AD  - Medizinische Klinik und Poliklinik IV, Ludwig-Maximilians-Universitat Munchen, 
      Munich, Germany.
FAU - Yuen, Kevin C J
AU  - Yuen KCJ
AD  - Swedish Pituitary Center, Swedish Neuroscience Institute, Seattle, WA, USA.
AD  - Barrow Neurological Institute, Phoenix, AZ, USA.
FAU - Roughton, Michael
AU  - Roughton M
AD  - Novartis Pharma AG, Basel, Switzerland.
FAU - Kriemler-Krahn, Ulrike
AU  - Kriemler-Krahn U
AD  - Novartis Pharma AG, Basel, Switzerland.
FAU - Tauchmanova, Libuse
AU  - Tauchmanova L
AD  - Novartis Pharma AG, Basel, Switzerland.
FAU - Maamari, Ricardo
AU  - Maamari R
AD  - Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
FAU - Giordano, Carla
AU  - Giordano C
AD  - Section of Endocrinology, Diabetology and Metabolism, Department of Health 
      Promotion Sciences Maternal and Infantile Care, Internal Medicine and Medical 
      Specialities (PROMISE), University of Palermo, Palermo, Italy.
LA  - eng
SI  - ClinicalTrials.gov/NCT02310269
PT  - Clinical Trial
PT  - Journal Article
PL  - United States
TA  - Pituitary
JT  - Pituitary
JID - 9814578
RN  - 51110-01-1 (Somatostatin)
RN  - 98H1T17066 (pasireotide)
SB  - IM
MH  - Adult
MH  - Female
MH  - Humans
MH  - Hyperglycemia/physiopathology
MH  - Male
MH  - Middle Aged
MH  - Multicenter Studies as Topic
MH  - Pituitary ACTH Hypersecretion/*drug therapy/physiopathology
MH  - Somatostatin/adverse effects/*analogs & derivatives/therapeutic use
MH  - Treatment Outcome
PMC - PMC6728293
OTO - NOTNLM
OT  - Cushing's disease
OT  - Hypercortisolism
OT  - Pasireotide
OT  - Pituitary
OT  - Safety
COIS- LM has served on Advisory Boards for Novartis. TD has received Honoraria and 
      Research Grants from, served on Advisory Boards for, and acted as a Consultant 
      for Novartis. KCJY has received Research Grants from Corcept Therapeutics and 
      Novartis and served on Advisory Boards and acted as a Consultant for Corcept 
      Therapeutics, Novartis, and Strongbridge. UKK, LT, MR, and RM are employees of 
      Novartis. CG has received Research Grants from and served on Advisory Boards for 
      Novartis. JS has received Lecture Fees from Novartis, Ipsen, and Pfizer and has 
      served on Advisory Boards for Novartis and Ipsen.
EDAT- 2019/08/24 06:00
MHDA- 2020/03/24 06:00
PMCR- 2019/08/22
CRDT- 2019/08/24 06:00
PHST- 2019/08/24 06:00 [pubmed]
PHST- 2020/03/24 06:00 [medline]
PHST- 2019/08/24 06:00 [entrez]
PHST- 2019/08/22 00:00 [pmc-release]
AID - 10.1007/s11102-019-00984-6 [pii]
AID - 984 [pii]
AID - 10.1007/s11102-019-00984-6 [doi]
PST - ppublish
SO  - Pituitary. 2019 Oct;22(5):542-551. doi: 10.1007/s11102-019-00984-6.