Topical Ocular Glucocorticoid Leads to Cushing’s Syndrome in 9-Year-Old

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Cushing’s Syndrome Etiology Affects Adrenal Function Recovery

The aim was to analyze the postsurgical duration of adrenal insufficiency of patients with Cushing’s disease (CD), adrenal CS and ectopic CS.

Design:

We performed a retrospective analysis based on the case records of 230 CS patients in our tertiary referral center treated from 1983 to 2014. The mean follow-up time was 8 years.

The probability of recovering adrenal function within a 5 years follow-up differed significantly between subtypes (p=0.001). It was 82 % in ectopic CS, 58 % in Cushing’s disease and 38 % in adrenal CS. In the total cohort with restored adrenal function (n=52) the median time to recovery differed between subtypes: 0.6 (IQR 0.03–1.1) years in ectopic CS, 1.4 (IQR 0.9–3.4) years in CD, and 2.5 (IQR 1.6–5.4) years in adrenal CS (p=0.002). In CD the Cox proportional-hazards model showed that the probability of recovery was associated with younger age (hazard ratio 0.896, 95% CI 0.822–0.976, p=0.012), independently of sex, BMI, duration of symptoms, and basal ACTH and cortisol levels. There was no correlation with length and extend of hypercortisolism or postoperative glucocorticoid replacement doses.

Conclusions:

Time to recovery of adrenal function is dependent on the underlying etiology of CS.

Adrenocortical Carcinoma and Pulmonary Embolism From Tumoral Extension

Adrenococortical carcinoma (ACC) is a rare cancer, occurring at the rate of one case in two million person years. Cushing syndrome or a mixed picture of excess androgen and glucocorticoid production are the most common presentations of ACC. Other uncommon presentations include abdominal pain and adrenal incidentalomas. In the present report, a 71-year-old male presented with abdominal pain and was eventually diagnosed with ACC. He was found to have pulmonary thromboembolism following an investigation for hypoxemia, with the tumor thrombus extending upto the right atrium. This interesting case represents the unique presentation of a rare tumor, which if detected late or left untreated is associated with poor outcomes, highlighting the need for a low index of suspicion for ACC when similar presentations are encountered in clinical practice.ACC is a rare but aggressive tumor. ACC commonly presents with rapid onset of hypercortisolism, combined hyperandrogenism and hypercortisolism, or uncommonly with compressive symptoms. Clinicians should have a low index of suspicion for ACC in patients presenting with rapid onset of symptoms related to hypercortisolism and/or hyperandrogenism. Venous thromboembolism and extension of the tumor thrombus to the right side of the heart is a very rare but serious complication of ACC that clinicans should be wary of. The increased risk of venous thromboembolism in ACC could be explained by direct tumor invasion, tumor thrombi or hypercoagulability secondary to hypercortisolism. Early diagnosis and prompt treatment can improve the long-term survival of patients with ACC.Endocrinology, diabetes & metabolism case reports. 2019 Nov 25 [Epub ahead of print]

Skand Shekhar, Sriram Gubbi, Georgios Z Papadakis, Naris Nilubol, Fady Hannah-Shmouni

Section on Endocrinology & Genetics, National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA., Diabetes, Endocrinology, and Obesity Branch, National Institute of Digestive and Kidney Diseases, National Institutes of Health, Bethesda, Maryland, USA., Department of Medical Imaging, Heraklion University Hospital, Medical School, University of Crete, Crete, Greece., Surgical Oncology Program, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USA.

 

From https://www.urotoday.com/recent-abstracts/urologic-oncology/adrenal-diseases/118539-adrenocortical-carcinoma-and-pulmonary-embolism-from-tumoral-extension.html

ISTURISA® (osilodrostat) Now Available in Canada for the Treatment of Cushing’s Disease

ISTURISA® (osilodrostat) is indicated for the treatment of adult patients with Cushing’s disease who have persistent or recurrent hypercortisolism after primary pituitary surgery and/or irradiation, or for whom pituitary surgery is not an option.1

TORONTO, Jan. 13, 2026 /CNW/ – Recordati Rare Diseases Canada Inc. announced today the Canadian product availability of ISTURISA® (osilodrostat) for the treatment of adult patients with Cushing’s disease who have persistent or recurrent hypercortisolism following pituitary surgery and/or irradiation, or for whom surgery is not an option.1 This is following the marketing authorisation of ISTURISA® in Canada on July 5, 2025.

Dr. André Lacroix, Professor of Medicine at the University of Montreal and internationally recognized authority in Cushing’s syndrome, commented on the importance of this new treatment option: ” ISTURISA® is an important addition to the treatment options for Cushing’s disease, a rare and debilitating condition. Achieving control of cortisol overproduction is an important strategy in helping patients manage Cushing’s disease.”

ISTURISA’s approval is supported by data from the LINC 3 and LINC 4 Phase III clinical studies, which demonstrated clinically meaningful reductions in mean urinary free cortisol (mUFC) levels and showed a favourable safety profile. ISTURISA® is available as 1 mg, 5 mg, and 10 mg film-coated tablets, enabling individualized titration based on cortisol levels and clinical response.1

About Cushing’s Disease

Cushing disease is a rare disorder of hypercortisolism caused by an adrenocorticotropic hormone (ACTH)-secreting pituitary adenoma, which in turn stimulates the adrenal glands to produce excess cortisol. Prolonged exposure to elevated cortisol levels is associated with substantial morbidity and mortality and impaired quality of life (QoL). Accordingly, normalization of cortisol is the primary treatment goal for Cushing disease.2

About Isturisa®

ISTURISA® is an inhibitor of 11β‐hydroxylase (CYP11B1), the enzyme responsible for the final step of cortisol synthesis in the adrenal gland. ISTURISA® is taken twice daily and is available as 1 mg, 5 mg and 10 mg film‐coated tablets, allowing for individualized titration based on cortisol levels and clinical response. For full prescribing information, healthcare professionals are encouraged to consult the Isturisa Product Monograph at https://recordatirarediseases.com/wp content/uploads/2025/08/ISTURISAProduct-Monograph-English-Current.pdf

Recordati Rare Diseases is Recordati’s dedicated business unit focused on rare diseases. Recordati is an international pharmaceutical Group listed on the Italian Stock Exchange (XMIL: REC), with roots dating back to a family-run pharmacy in Northern Italy in the 1920s. Our fully integrated operations span clinical development, chemical and finished product manufacturing, commercialisation and licensing. We operate in approximately 150 countries across EMEA, the Americas and APAC with over 4,500 employees.

Recordati Rare Diseases’ mission is to reduce the impact of extremely rare and devastating diseases by providing urgently needed therapies. We work side-by-side with rare disease communities to increase awareness, improve diagnosis and expand availability of treatments for people with rare diseases.

Recordati Rare Diseases Canada Inc. is the company’s Canada offices located inToronto, Ontario, with the North America headquarter offices located in New Jersey, US, and the global headquarter offices located in Milan, Italy.

This document contains forward-looking statements relating to future events and future operating, economic and financial results of the Recordati group. By their nature, forward-looking statements involve risk and uncertainty because they depend on the occurrence of future events and circumstances. Actual results may therefore differ materially from those forecast for a variety of reasons, most of which are beyond the Recordati group’s control. The information on the pharmaceutical specialties and other products of the Recordati group contained in this document is intended solely as information on the activities of the Recordati Group, and, as such, it is not intended as a medical scientific indication or recommendation, or as advertising.

References:
1. Isturisa® Product Monograph. 2025-07-03
2. Gadelha M et al. J Clin Endocrinol Metab. 2022 Jun 16;107(7): e2882-e2895

SOURCE Recordati Rare Diseases Canada Inc.

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Media Relations: spPR Inc., Sonia Prashar, 416.560.6753, Soniaprashar@sppublicrelations.com

https://www.newswire.ca/news-releases/isturisa-r-osilodrostat-now-available-in-canada-for-the-treatment-of-cushing-s-disease-879473023.html

Transsphenoidal Surgery Leads to Remission in Children with Cushing’s Disease

Transsphenoidal surgery — a minimally invasive surgery for removing pituitary tumors in Cushing’s disease patients — is also effective in children and adolescents with the condition, leading to remission with a low rate of complications, a study reports.

The research, “Neurosurgical treatment of Cushing disease in pediatric patients: case series and review of literature,” was published in the journal Child’s Nervous System.

Transsphenoidal (through the nose) pituitary surgery is the main treatment option for children with Cushing’s disease. It allows the removal of pituitary adenomas without requiring long-term replacement therapy, but negative effects on growth and puberty have been reported.

In the study, a team from Turkey shared its findings on 10 children and adolescents (7 females) with the condition, who underwent microsurgery (TSMS) or endoscopic surgery (ETSS, which is less invasive) — the two types of transsphenoidal surgery.

At the time of surgery, the patients’ mean age was 14.8 years, and they had been experiencing symptoms for a mean average of 24.2 months. All but one had gained weight, with a mean body mass index of 29.97.

Their symptoms included excessive body hair, high blood pressure, stretch marks, headaches, acne, “moon face,” and the absence of menstruation.

The patients were diagnosed with Cushing’s after their plasma cortisol levels were measured, and there was a lack of cortical level suppression after they took a low-dose suppression treatment. Measurements of their adrenocorticotropic (ACTH) hormone levels then revealed the cause of their disease was likely pituitary tumors.

Magnetic resonance imaging (MRI) scans, however, only enabled tumor localization in seven patients: three with a microadenoma (a tumor smaller than 10 millimeters), and four showed a macroadenoma.

CD diagnosis was confirmed by surgery and the presence of characteristic pituitary changes. The three patients with no sign of adenoma on their MRIs showed evidence of ACTH-containing adenomas on tissue evaluation.

Eight patients underwent TSMS, and 2 patients had ETSS, with no surgical complications. The patients were considered in remission if they showed clinical adrenal insufficiency and serum cortisol levels under 2.5 μg/dl 48 hours after surgery, or a cortisol level lower than 1.8 μg/dl with a low-dose dexamethasone suppression test at three months post-surgery. Restoration of normal plasma cortisol variation, eased symptoms, and no sign of adenoma in MRI were also requirements for remission.

Eight patients (80%) achieved remission, 4 of them after TSMS. Two patients underwent additional TSMS for remission. Also, 1 patient had ETSS twice after TSMS to gain remission, while another met the criteria after the first endoscopic surgery.

The data further showed that clinical recovery and normalized biochemical parameters were achieved after the initial operation in 5 patients (50%). Three patients (30%) were considered cured after additional operations.

The mean cortisol level decreased to 8.71 μg/dl post-surgery from 23.435 μg/dl pre-surgery. All patients were regularly evaluated in an outpatient clinic, with a mean follow-up period of 11 years.

Two patients showed pituitary insufficiency. Also, 2 had persistent hypocortisolism — too little cortisol — one of whom also had diabetes insipidus, a disorder that causes an imbalance of water in the body. Radiotherapy was not considered in any case.

“Transsphenoidal surgery remains the mainstay therapy for CD [Cushing’s disease] in pediatric patients as well as adults,” the scientists wrote. “It is an effective treatment option with low rate of complications.”

 

From https://cushingsdiseasenews.com/2019/01/15/transsphenoidal-surgery-enables-cushings-disease-remission-pediatric-patients-study/