A Phase 2 Study of Continuous Subcutaneous Hydrocortisone Infusion in Adults With Congenital Adrenal Hyperplasia

Abstract

CSHI is a safe and well-tolerated modality of cortisol replacement that effectively approximates physiologic cortisol secretion in patients with classic CAH poorly controlled on conventional therapy. Improved adrenal steroid control and positive effects on HRQoL suggest that CSHI should be considered a treatment option for classic CAH. The long-term effect on established comorbidities requires further study.

Long‐term use of continuous subcutaneous hydrocortisone infusion therapy in patients with congenital adrenal hyperplasia

“…Two patients switched back to oral glucocorticoid therapy as they found CSHI therapy did not suit their lifestyle. Details of the study participants and baseline clinical and hormonal characteristics have been previously described . One patient underwent bariatric surgery for morbid obesity and was excluded from this study; however, she currently is maintained on CSHI therapy .…”

“…The objective of this study was to assess the long‐term safety and efficacy of CSHI therapy. Specifically, our goal was to observe whether the improvements achieved in androgen control and HRQoL estimates after 6 months of CSHI therapy were maintained at 18 months . All patients remained on their daily fludrocortisone replacement, and dose adjustments were made if necessary based on plasma renin activity levels and clinical symptomatology.…”

“…Novel glucocorticoid replacement approaches aiming to replace cortisol in a physiological manner are under study, including the use of a modified‐release formulation of hydrocortisone (Chronocort ® ) and continuous subcutaneous hydrocortisone infusion (CSHI) . We previously reported improvements in adrenal androgen control and health‐related quality‐of‐life (HRQoL) estimates with the use of CSHI therapy in adults with difficult‐to‐treat CAH, defined as having androgen‐ and glucocorticoid‐related comorbidities on conventional oral glucocorticoid therapy . Overall, short‐term (6 months) use of CSHI was found to be safe, effective and a well‐tolerated method of replacing cortisol in adults with classic CAH with improved outcomes; positive effects on HRQoL were the most significant .…”

“…We previously reported improvements in adrenal androgen control and health‐related quality‐of‐life (HRQoL) estimates with the use of CSHI therapy in adults with difficult‐to‐treat CAH, defined as having androgen‐ and glucocorticoid‐related comorbidities on conventional oral glucocorticoid therapy . Overall, short‐term (6 months) use of CSHI was found to be safe, effective and a well‐tolerated method of replacing cortisol in adults with classic CAH with improved outcomes; positive effects on HRQoL were the most significant . The aim of this study was to evaluate the safety and efficacy of long‐term use of CSHI.…”

Klassisk medfødt binyrebarkhyperplasi

“…Denne behandlingen gjenskaper dessverre ikke den normale døgnprofilen av kortisol, og medikamenter med modifisert frisetting av hydrokortison er under utvikling (7). Kontinuerlig subkutan hydrokortisoninfusjon kan vaere nyttig hos enkelte pasienter (8).…”