scholarly journals Infantile idiopathic intracranial hypertension: case report and review of the literature

2022 ◽  
Vol 48 (1) ◽  
Author(s):  
Francesco Del Monte ◽  
Laura Bucchino ◽  
Antonia Versace ◽  
Irene Tardivo ◽  
Emanuele Castagno ◽  
...  
Keyword(s):  
Cerebrospinal Fluid ◽  
Intracranial Hypertension ◽  
Lumbar Puncture ◽  
Idiopathic Intracranial Hypertension ◽  
Symptom Relief ◽  
Clinical Manifestations ◽  
Initial Symptom ◽  
Anterior Fontanel ◽  
Opening Pressure ◽  
Cerebral Mri

Abstract Background Idiopathic intracranial hypertension is an infrequent condition of childhood, and is extremely rare in infants, with only 26 cases described. The etiology is still unknown. Typical clinical manifestations change with age, and symptoms are atypical in infants, thus the diagnosis could be late. This is based on increased opening pressure at lumbar puncture, papilloedema and normal cerebral MRI. The measurement of cerebrospinal fluid opening pressure in infants is an issue because many factors may affect it, and data about normal values are scanty. The mainstay of treatment is acetazolamide, which allows to relieve symptoms and to avoid permanent visual loss if promptly administered. Case presentation We report the case of an 8-month-old infant admitted because of vomit, loss of appetite and irritability; later, also bulging anterior fontanel was observed. Cerebral MRI and cerebrospinal fluid analysis resulted negative and after two lumbar punctures he experienced initial symptom relief. Once the diagnosis of idiopathic intracranial hypertension was made, he received oral acetazolamide, and corticosteroids, with progressive symptom resolution. Conclusions Infantile idiopathic intracranial hypertension is extremely rare, and not well described yet. Bulging anterior fontanel in otherwise healthy infants with normal neuroimaging should be always considered suggestive, but can be a late sign, while irritability and anorexia, especially if associated with vomiting, may represent an early sign. In such cases, lumbar puncture should be always done, hopefully with cerebrospinal fluid opening pressure measurement, which is among coded diagnostic criteria, but whose threshold is controversial in infants. Early diagnosis, timely treatment and strict follow-up help to prevent vision loss or death of affected infants.

scholarly journals Benign Intracranial Hypertension Due to Hypoparathyroidism: A Case Report

2022 ◽  
Vol 12 ◽  
Author(s):  
Giorgia Sforza ◽  
Annalisa Deodati ◽  
Romina Moavero ◽  
Laura Papetti ◽  
Ilaria Frattale ◽  
...  
Keyword(s):  
Cerebrospinal Fluid ◽  
Case Report ◽  
Intracranial Hypertension ◽  
Lumbar Puncture ◽  
Idiopathic Intracranial Hypertension ◽  
Clinical Manifestations ◽  
Nausea And Vomiting ◽  
Benign Intracranial Hypertension ◽  
Blood Tests ◽  
Organ Systems

Objective: The objective of this study is to present the rare case of a young girl with idiopathic intracranial hypertension secondary to hypoparathyroidism.Background: Idiopathic intracranial hypertension is a neurological syndrome characterized by elevated intracranial pressure (> 25 cmH2O) in the absence of intracerebral abnormalities or hydrocephalus. The pathophysiology of idiopathic intracranial hypertension is unknown, and rare cases of idiopathic intracranial hypertension secondary to hypoparathyroidism have been described. It is supposed that hypocalcemia causes decrease in the absorption of cerebrospinal fluid in arachnoidal granulations.Methods: The workup of the girl with idiopathic intracranial hypertension and hypoparathyroidism included physical examination, blood tests, diagnostic imaging, and lumbar puncture.Results: We present a 9-year-old female patient who was hospitalized for headache associated with nausea and vomiting for 3 weeks. She underwent an ophthalmologic examination that revealed papilledema. Lumbar puncture revealed an opening pressure of 65 cm H2O; cerebrospinal fluid analysis and brain computed tomography scan were normal. The patient started taking acetazolamide. Blood tests revealed hypocalcemia associated with high phosphorus level and undetectable PTH hormone, which led us to suspect hypoparathyroidism. She had never had cramps, paraesthesias, or tetany. Chvostek's and Trousseau's signs were positive. In the neck ultrasonography, parathyroids were not visible. Oral supplementation with calcitriol and calcium was started. Headache, nausea, and vomiting immediately disappeared after the lumbar puncture, and the papilledema improved gradually.Conclusions: Several anecdotal cases of idiopathic intracranial hypertension secondary to hypoparathyroidism have been described. However, our case report is of particular interest, since the child did not present with typical neurological hypoparathyroidism symptoms. Therefore, we recommend that hypoparathyroidism should be included in diagnostic investigations on children with clinical findings of idiopathic intracranial hypertension, because clinical manifestations of hypoparathyroidism are variable and may involve almost all organ systems.

scholarly journals 11β-Hydroxysteroid dehydrogenase type 1 inhibition in idiopathic intracranial hypertension: a double-blind randomized controlled trial

2020 ◽  
Vol 2 (1) ◽  
Author(s):  
Keira Markey ◽  
James Mitchell ◽  
Hannah Botfield ◽  
Ryan S Ottridge ◽  
Tim Matthews ◽  
...  
Keyword(s):  
Intracranial Hypertension ◽  
Lumbar Puncture ◽  
Idiopathic Intracranial Hypertension ◽  
Controlled Trial ◽  
Hydroxysteroid Dehydrogenase ◽  
Opening Pressure ◽  
Double Blind ◽  
Randomized Controlled

Abstract Treatment options for idiopathic intracranial hypertension are limited. The enzyme 11β-hydroxysteroid dehydrogenase type 1 has been implicated in regulating cerebrospinal fluid secretion, and its activity is associated with alterations in intracranial pressure in idiopathic intracranial hypertension. We assessed therapeutic efficacy, safety and tolerability and investigated indicators of in vivo efficacy of the 11β-hydroxysteroid dehydrogenase type 1 inhibitor AZD4017 compared with placebo in idiopathic intracranial hypertension. A multicenter, UK, 16-week phase II randomized, double-blind, placebo-controlled trial of 12-week treatment with AZD4017 or placebo was conducted. Women aged 18–55 years with active idiopathic intracranial hypertension (>25 cmH2O lumbar puncture opening pressure and active papilledema) were included. Participants received 400 mg of oral AZD4017 twice daily compared with matching placebo over 12 weeks. The outcome measures were initial efficacy, safety and tolerability. The primary clinical outcome was lumbar puncture opening pressure at 12 weeks analysed by intention-to-treat. Secondary clinical outcomes were symptoms, visual function, papilledema, headache and anthropometric measures. In vivo efficacy was evaluated in the central nervous system and systemically. A total of 31 subjects [mean age 31.2 (SD = 6.9) years and body mass index 39.2 (SD = 12.6) kg/m2] were randomized to AZD4017 (n = 17) or placebo (n = 14). At 12 weeks, lumbar puncture pressure was lower in the AZD4017 group (29.7 cmH2O) compared with placebo (31.3 cmH2O), but the difference between groups was not statistically significant (mean difference: −2.8, 95% confidence interval: −7.1 to 1.5; P = 0.2). An exploratory analysis assessing mean change in lumbar puncture pressure within each group found a significant decrease in the AZD4017 group [mean change: −4.3 cmH2O (SD = 5.7); P = 0.009] but not in the placebo group [mean change: −0.3 cmH2O (SD = 5.9); P = 0.8]. AZD4017 was safe, with no withdrawals related to adverse effects. Nine transient drug-related adverse events were reported. One serious adverse event occurred in the placebo group (deterioration requiring shunt surgery). In vivo biomarkers of 11β-hydroxysteroid dehydrogenase type 1 activity (urinary glucocorticoid metabolites, hepatic prednisolone generation, serum and cerebrospinal fluid cortisol:cortisone ratios) demonstrated significant enzyme inhibition with the reduction in serum cortisol:cortisone ratio correlating significantly with reduction in lumbar puncture pressure (P = 0.005, R = 0.70). This is the first phase II randomized controlled trial in idiopathic intracranial hypertension evaluating a novel therapeutic target. AZD4017 was safe and well tolerated and inhibited 11β-hydroxysteroid dehydrogenase type 1 activity in vivo. Reduction in serum cortisol:cortisone correlated with decreased intracranial pressure. Possible clinical benefits were noted in this small cohort. A longer, larger study would now be of interest.

Lumbar puncture rapidly improves olfaction in patients with idiopathic intracranial hypertension: A cohort study

Cephalalgia ◽  
2020 ◽  
Vol 40 (5) ◽  
pp. 429-436 ◽  
Author(s):  
Nils J Becker ◽  
Sören Enge ◽  
Katharina Maria Kreutz ◽  
Felix Schmidt ◽  
Lutz Harms ◽  
...  
Keyword(s):  
Cerebrospinal Fluid ◽  
Intracranial Hypertension ◽  
Lumbar Puncture ◽  
Idiopathic Intracranial Hypertension ◽  
Fluid Pressure ◽  
Olfactory Function ◽  
Clinical Feature ◽  
Healthy Controls ◽  
Cerebrospinal Fluid Drainage ◽  
Olfactory Impairment

Background A lumbar puncture constitutes an important diagnostic procedure in the evaluation of idiopathic intracranial hypertension. Chronic overflow of cerebrospinal fluid into the sheaths of the olfactory nerves appears to be related to olfactory impairment in these patients. Here, we asked whether cerebrospinal fluid drainage in idiopathic intracranial hypertension patients improves olfactory function. Methods Fourteen idiopathic intracranial hypertension patients and 14 neurologic control patients were investigated before and after lumbar puncture using the extended Sniffin’ Sticks procedure. We assessed odor threshold, discrimination, and identification. In idiopathic intracranial hypertension patients, cerebrospinal fluid was drained until cerebrospinal fluid pressure had normalized. In addition, a third group of 14 healthy controls participated in the two smell tests at similar intervals. Results Relative to healthy controls, threshold, discrimination, and identification composite scores before lumbar puncture were significantly lower in idiopathic intracranial hypertension patients and also in neurologic controls. Following lumbar puncture, threshold, discrimination, and identification scores for neurologic controls remained unchanged whereas idiopathic intracranial hypertension patients showed robust improvement on the composite score as well as on all three subscores (all changes: p < 0.003), quickly regaining olfactory function in the normal range. Cerebrospinal fluid opening pressure was significantly correlated with improvement in threshold, discrimination, and identification score upon cerebrospinal fluid drainage ( r = 0.609, p = 0.021). Conclusion Olfactory impairment is an important, yet underappreciated, clinical feature of idiopathic intracranial hypertension. Lowering of increased intracranial pressure improves hyposmia. Our findings shed new light on the pathophysiology of cerebrospinal fluid circulation in idiopathic intracranial hypertension.

scholarly journals Therapeutic lumbar puncture for headache in idiopathic intracranial hypertension: Minimal gain, is it worth the pain?

Cephalalgia ◽  
2018 ◽  
Vol 39 (2) ◽  
pp. 245-253 ◽  
Author(s):  
Andreas Yiangou ◽  
James Mitchell ◽  
Keira Annie Markey ◽  
William Scotton ◽  
Peter Nightingale ◽  
...  
Keyword(s):  
Intracranial Hypertension ◽  
Lumbar Puncture ◽  
Idiopathic Intracranial Hypertension ◽  
Rating Scale ◽  
Acute Effect ◽  
Numeric Rating Scale ◽  
Severe Exacerbation ◽  
Opening Pressure ◽  
Headache Severity ◽  
Numeric Rating

Background Headache is disabling and prevalent in idiopathic intracranial hypertension. Therapeutic lumbar punctures may be considered to manage headache. This study evaluated the acute effect of lumbar punctures on headache severity. Additionally, the effect of lumbar puncture pressure on post-lumbar puncture headache was evaluated. Methods Active idiopathic intracranial hypertension patients were prospectively recruited to a cohort study, lumbar puncture pressure and papilloedema grade were noted. Headache severity was recorded using a numeric rating scale (NRS) 0–10, pre-lumbar puncture and following lumbar puncture at 1, 4 and 6 hours and daily for 7 days. Results Fifty two patients were recruited (mean lumbar puncture opening pressure 32 (28–37 cmCSF). At any point in the week post-lumbar puncture, headache severity improved in 71% (but a small reduction of −1.1 ± 2.6 numeric rating scale) and exacerbated in 64%, with 30% experiencing a severe exacerbation ≥ 4 numeric rating scale. Therapeutic lumbar punctures are typically considered in idiopathic intracranial hypertension patients with severe headaches (numeric rating scale ≥ 7). In this cohort, the likelihood of improvement was 92% (a modest reduction of headache pain by −3.0 ± 2.8 numeric rating scale, p = 0.012, day 7), while 33% deteriorated. Idiopathic intracranial hypertension patients with mild (numeric rating scale 1–3) or no headache (on the day of lumbar puncture, prior to lumbar puncture) had a high risk of post- lumbar puncture headache exacerbation (81% and 67% respectively). Importantly, there was no relationship between lumbar puncture opening pressure and headache response after lumbar puncture. Conclusion Following lumbar puncture, the majority of idiopathic intracranial hypertension patients experience some improvement, but the benefit is small and post-lumbar puncture headache exacerbation is common, and in some prolonged and severe. Lumbar puncture pressure does not influence the post-lumbar puncture headache.

Infantile Idiopathic Intracranial Hypertension: A Case Study and Review of the Literature

2019 ◽  
Vol 34 (13) ◽  
pp. 806-814 ◽  
Author(s):  
Sama Boles ◽  
Claudia Martinez-Rios ◽  
Daniel Tibussek ◽  
Daniela Pohl
Keyword(s):  
Cerebrospinal Fluid ◽  
Intracranial Hypertension ◽  
Lumbar Puncture ◽  
Idiopathic Intracranial Hypertension ◽  
Treatment Guidelines ◽  
Fluid Pressure ◽  
Specific Treatment ◽  
Unknown Etiology ◽  
Age Group ◽  
Review Of The Literature

Idiopathic intracranial hypertension, or pseudotumor cerebri, is an increase in cerebrospinal fluid pressure of unknown etiology. It is mostly seen in adults, less frequently in adolescents, rarely in younger children. Only 5 infants meeting idiopathic intracranial hypertension criteria have been mentioned in the literature. We report a case of a previously healthy 9-month-old boy who presented with irritability, decreased appetite, and a bulging fontanelle. Computed tomography (CT) head imaging and cerebrospinal fluid studies revealed normal results. The patient’s symptoms transiently resolved after the initial lumbar puncture, but 11 days later, his fontanelle bulged again. A second lumbar puncture revealed an elevated opening pressure of 35 cmH2O and led to a diagnosis of idiopathic intracranial hypertension in accordance with the modified Dandy Criteria. Treatment with acetazolamide at a dose of 25 mg/kg/d was initiated and the patient remained symptom-free for 6 weeks, followed by another relapse. His acetazolamide dose was increased to 37 mg/kg/d, with no further relapses to date. A diagnosis of idiopathic intracranial hypertension is challenging in infants, because the patients cannot yet verbalize typical idiopathic intracranial hypertension–related symptoms such as positional headaches, diplopia, or pulsatile tinnitus. Furthermore, it is more difficult to assess papilledema in that age group. If undetected and untreated, idiopathic intracranial hypertension may result in permanent visual deficits. Little is known about idiopathic intracranial hypertension in infants, and age-specific treatment guidelines are lacking. We discuss this rare case of infantile idiopathic intracranial hypertension and provide a review of the literature, including an overview of disease characteristics and outcomes of idiopathic intracranial hypertension in this very young age group.

scholarly journals Chiari I - Idiopathic Intracranial Hypertension Association After Failed Posterior Fossa Surgery. Case Report

2019 ◽  
Vol 30 (3) ◽  
pp. 252-260
Author(s):  
Felix Pastor Escartín ◽  
Vicent Quilis Quesada ◽  
Pau Capilla Guasch ◽  
Diego Tabarés Palacín ◽  
Esteban Vega Torres ◽  
...  
Keyword(s):  
Intracranial Hypertension ◽  
Lumbar Puncture ◽  
Posterior Fossa ◽  
Idiopathic Intracranial Hypertension ◽  
Decompression Surgery ◽  
Posterior Fossa Decompression ◽  
Type I ◽  
Posterior Fossa Surgery ◽  
Opening Pressure ◽  
Chiari I

Objectives: Several papers have been published relating the Idiopathic Intracranial Hypertension Syndrome (HTII) to the Arnold Chiari type I malformation (AC1M). Both entities have clinical and demographic similarities, a poorly defined etiology and, sometimes common therapeutic posibilities. A correlation between both entities has been suggested, especially in a subgroup of patients in whom posterior fossa decompression surgery fails. With regard to a case, we reviewed the literature and proposed our hypothesis about the origin of Chiari-HTII syndrome and its therapeutic possibilities. Case presentation: A 41year-old patient with mild obesity, menstrual abnormalities and empty Sella Turcicae, was operated on with an AC1M associating basilar impression and syringomyelia causing all together a centromedullary syndrome. After posterior fossa decompression surgery and successful arthrodesis, she improved in the immediate postoperative period. Nevertheless, she soon developed symptoms of intracranial hypertension (ICH), and showed increased opening pressure in lumbar puncture compatible with HTII syndrome. A ventriculoperitoneal shunt (VPS) was implanted with clinical improvement and 12 months later the syringomyelia was absent on in the magnetic resonance (MRI). Conclusion: The Chiari I-HTII syndrome is described as the coexistence of ICH symptoms after failed posterior fossa surgery, in patients with no flow MRI anomalies, and increased opening pressure at the lumbar puncture. In our experience, both entities seem to overlap in a common syndrome and must be taken into account, especially in patients with atypical onset of symptoms or patients in whom conservative treatment fails.

Co-occurrence of acute ophthalmoplegia (without ataxia) and idiopathic intracranial hypertension

2018 ◽  
Vol 29 (4) ◽  
pp. NP1-NP4 ◽  
Author(s):  
Justin Yeak ◽  
Mimiwati Zahari ◽  
Sujaya Singh ◽  
Nor Fadhilah Mohamad
Keyword(s):  
Intracranial Pressure ◽  
Intracranial Hypertension ◽  
Lumbar Puncture ◽  
Idiopathic Intracranial Hypertension ◽  
Miller Fisher Syndrome ◽  
Upper Respiratory Tract ◽  
Raised Intracranial Pressure ◽  
Opening Pressure ◽  
Limb Weakness ◽  
Fisher Syndrome

Background: Acute ophthalmoparesis without ataxia was designated as ‘atypical Miller Fisher syndrome’ as it presents with progressive, relatively symmetrical ophthalmoplegia, but without ataxia nor limb weakness, in the presence of anti-GQ1b antibody. Idiopathic intracranial hypertension is characterized by signs of raised intracranial pressure occurring in the absence of cerebral pathology, with normal composition of cerebrospinal fluid and a raised opening pressure of more than 20 cmH2O during lumbar puncture. We aim to report a rare case of acute ophthalmoplegia with co-occurrence of raised intracranial pressure. Case Description: A 28-year-old gentleman with body mass index of 34.3 was referred to us for management of double vision of 2 weeks duration. His symptom started after a brief episode of upper respiratory tract infection. His best corrected visual acuity was 6/6 OU. He had bilateral sixth nerve palsy worse on the left eye and bilateral hypometric saccade. His deep tendon reflexes were found to be hyporeflexic in all four limbs. No sensory or motor power deficit was detected, and his gait was normal. Plantar reflexes were downwards bilaterally and cerebellar examination was normal. Both optic discs developed hyperaemia and swelling. Magnetic resonance imaging of brain was normal and lumbar puncture revealed an opening pressure of 50 cmH2O. Anti-GQ1b IgG and anti-GT1a IgG antibody were tested positive. Conclusion: Acute ophthalmoparesis without ataxia can present with co-occurrence of raised intracranial pressure. It is important to have a full fundoscopic assessment to look for papilloedema in patients presenting with Miller Fisher syndrome or acute ophthalmoparesis without ataxia.

scholarly journals Clinical Manifestations, Neuroimaging Findings, and Treatment of Idiopathic Intracranial Hypertension in a Nepalese Tertiary Centre

2021 ◽  
Vol 4 (2) ◽  
pp. 468-472
Author(s):  
Niraj Gautam ◽  
Rajeev Ojha ◽  
Sanjeev Kharel ◽  
Bikram Prasad Gajurel ◽  
Sanjeeta Sitaula ◽  
...  
Keyword(s):  
Visual Impairment ◽  
Intracranial Hypertension ◽  
Idiopathic Intracranial Hypertension ◽  
Clinical Manifestations ◽  
Transverse Sinus ◽  
Empty Sella ◽  
University Teaching ◽  
Opening Pressure ◽  
Ventriculoperitoneal Shunting ◽  
Tertiary Center

Introduction: Idiopathic Intracranial Hypertension is characterized by headache, visual impairment, papilledema, and increased cerebrospinal fluid opening pressure. We aim to evaluate clinical manifestations, neuroimaging findings, and treatment of Idiopathic Intracranial Hypertension in a Nepalese tertiary center. Materials and Methods: We retrospectively included patients with the diagnosis of Idiopathic Intracranial Hypertension who were admitted to the Neurology department of Tribhuvan University Teaching Hospital from 2019 June to 2021 May and presented to the Neuro-ophthalmology outpatient clinic of the hospital for follow-up. Results: Out of 16 Idiopathic Intracranial Hypertension patients, 12(75%) patients had either headache or ocular pain.  Reduced visual acuity and progressive visual loss were found in 44% of patients. Normal CSF opening pressure was found in 19%, 44% had CSF opening pressure at a range of 20-30 cm H20 and > 30 cm H20 in 37.5%. 15 Idiopathic Intracranial Hypertension patients (93.7%) were subtyped as Typical and 1 patient was diagnosed as Fulminant Idiopathic Intracranial Hypertension. Acetazolamide was used for treatment in 15 patients and 1 patient needed ventriculoperitoneal shunting. 56% Seven patients (44%) had some abnormal findings: partial empty sella (44%), and tortuous optic nerve (31%), flattened posterior eyeball (31%), and hypoplastic transverse sinus (19%). Conclusions: Idiopathic Intracranial Hypertension is an uncommon diagnosis but should be suspected in patients with chronic headaches with visual impairment. In low-resource settings, proper history along with neurological and ophthalmological examinations can even detect the early features and timely referral can save the vision and disability of Idiopathic Intracranial Hypertension patients.

scholarly journals 11β-Hydroxysteroid Dehydrogenase Type 1 inhibition in Idiopathic Intracranial Hypertension: a double-blind randomized controlled trial

2019 ◽  
Author(s):  
Keira Markey ◽  
James Mitchell ◽  
Hannah Botfield ◽  
Ryan S Ottridge ◽  
Tim Matthews ◽  
...  
Keyword(s):  
Intracranial Hypertension ◽  
Lumbar Puncture ◽  
Idiopathic Intracranial Hypertension ◽  
Controlled Trial ◽  
Hydroxysteroid Dehydrogenase ◽  
Opening Pressure ◽  
Double Blind ◽  
Randomized Controlled

AbstractTreatment options for idiopathic intracranial hypertension are limited. The enzyme 11β-hydroxysteroid dehydrogenase type 1 has been implicated in regulating cerebrospinal fluid secretion, and its activity is associated with alterations in intracranial pressure in idiopathic intracranial hypertension. We assessed therapeutic efficacy, safety and tolerability, and investigate indicators of in vivo efficacy of the 11β-hydroxysteroid dehydrogenase type 1 inhibitor AZD4017 compared to placebo in idiopathic intracranial hypertension. A multicenter, UK, 16-week phase II randomized, double-blind, placebo-controlled trial of 12-weeks treatment with AZD4017 or placebo was conducted. Women aged 18 to 55 years with active idiopathic intracranial hypertension (>25cmH2O lumbar puncture opening pressure and active papilledema) were included. Participants received 400mg twice daily of oral AZD4017 compared to matching placebo over 12-weeks. The outcome measures were initial efficacy, safety and tolerability. The primary clinical outcome was lumbar puncture opening pressure at 12 weeks analysed by intention-to-treat. Secondary clinical outcomes were symptoms, visual function, papilledema, headache and anthropological measures. In vivo efficacy was evaluated in the central nervous system and systemically. 31 subjects (mean age 31.2 (SD=6.9) years and BMI 39.2 (SD=12.6) kg/m2) were randomized to AZD4017 (n=17) or placebo (n=14). At 12 weeks, lumbar puncture pressure was lower in the AZD4017 group (29.7 cmH2O) compared with placebo (31.3 cmH2O), but the difference between groups was not statistically significant (mean difference: −2.8, 95% confidence interval: −7.1-1.5; p=0.2). An exploratory analysis assessing mean change in lumbar puncture pressure within each group found a significant decrease in the AZD4017 group (mean change: −4.3 cmH2O (SD=5.7); p=0.009) but not in the placebo group (mean change: −0.3 cmH2O (SD=5.9); p=0.8). AZD4017 was safe, with no withdrawals related to adverse effects. Nine transient drug-related adverse events were reported. One serious adverse event occurred in the placebo group (deterioration requiring shunt surgery). In vivo biomarkers of 11β-hydroxysteroid dehydrogenase type 1 activity (urinary glucocorticoid metabolites, hepatic prednisolone generation and CSF cortisone to cortisol ratios) demonstrated significant enzyme inhibition. This is the first phase 2 randomized controlled trial in idiopathic intracranial hypertension evaluating a novel therapeutic target. AZD4017 was safe, well-tolerated and inhibited 11β-hydroxysteroid dehydrogenase type 1 activity in vivo. Possible clinical benefits were noted in this small cohort. A longer, larger study would now be of interest.

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