Pituitary & hypothalamus

Discontinuation of Drug Treatment in Cushing's Disease Not Cured by Pituitary Surgery

J Clin Endocrinol MetabPublished Retrospective cohort study

Authors' conclusion

DT allowed at least 1 normal UFC in 23/29 patients (79%) but obtained long-term control in only 11/29 (38%), as discontinuation rate was high, although similar to published data. Interestingly, a successful 2nd TSS was the cause for discontinuing efficient and well-tolerated DT in 5 patients. Further studies will show whether different strategies with cortisol synthesis inhibitors may allow for a lower discontinuation rate in patients not candidates for a 2nd TSS so that BA may be avoided in these patients.

Study context

Population
Patients with Cushing's disease (CD) not cured by first transsphenoidal surgery (TSS) (19 TSS failure, 17 recurrence), out of 119 patients with CD treated by a first TSS; average follow-up 6.1 years
Participants
29 / 16 / 14 / 5
Exposure
Drug treatment (DT) vs 2nd TSS vs bilateral adrenalectomy (BA) vs pituitary radiotherapy (PR)
  • DTn = 29
  • 2nd TSSn = 16
  • BAn = 14
  • PRn = 5
Outcome
Long-term continuation of DT; control defined as normalization of UFC, final treatment (FT) as the treatment allowing control at last follow-up
Follow-up
Last
Methodological detailssingle-centre, centre prioritizing 2nd TSS
  • Single-centre
  • Centre prioritizing 2nd TSS

Main finding

As reported in the abstract

GroupValueInitiatedAt least 1 normal urinary free cortisol (UFC)Discontinued before last follow-upFinal treatment (FT)
DT38%29/36 (81%)23/29 (79%)18/29 (62%)11/29 (38%)
2nd TSS50%8/16 (50%)
BA100%14/14 (100%)
PR0 events0/5

Value

DT38%
2nd TSS50%
BA100%
PR0 events

Initiated

DT29/36 (81%)
2nd TSS— not stated in the abstract
BA— not stated in the abstract
PR— not stated in the abstract

At least 1 normal urinary free cortisol (UFC)

DT23/29 (79%)
2nd TSS— not stated in the abstract
BA— not stated in the abstract
PR— not stated in the abstract

Discontinued before last follow-up

DT18/29 (62%)
2nd TSS— not stated in the abstract
BA— not stated in the abstract
PR— not stated in the abstract

Final treatment (FT)

DT11/29 (38%)
2nd TSS8/16 (50%)
BA14/14 (100%)
PR0/5
Control achieved (all patients)
33/36 (92%)

Observational data: associations, not effects.

Further findings

Discontinuation of DT in published CD prospective clinical trials

Discontinuation at 1 year
11% to 51%
Discontinuation before 5 years
32% to 74%

Parts of this Brief are withheld: the automatic checks could not confirm them against the abstract.

Original publication

Discontinuation of Drug Treatment in Cushing's Disease Not Cured by Pituitary Surgery. J Clin Endocrinol Metab. Published 14 November 2023. doi:10.1210/clinem/dgad662

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