Papilledema and hypervitaminosis A after elexacaftor/tezacaftor/ivacaftor for cystic fibrosis

Miller, Matthew J.; Foroozan, Rod

doi:10.1016/j.jcjo.2021.04.018

Cited by 22 publications

(18 citation statements)

References 10 publications

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“… 8 However, 1 of our patients who had a congenital cranial malformation had severe intracranial hypertension and died. There are some case reports of severe intracranial hypertension following ELX/TEZ/IVA treatment, mainly related to hypervitaminosis A toxicity, 10 , 11 but with good prognosis despite continuation of treatment with ELX/TEZ/IVA, supporting our belief that our patient’s unknown congenital malformation was determinant for the negative outcome.…”

Section: Discussionsupporting

confidence: 79%

“…(8) No entanto, 1 de nossos pacientes com malformação craniana congênita apresentou hipertensão intracraniana grave e faleceu. Existem alguns relatos de casos de hipertensão intracraniana grave após tratamento com ELX/TEZ/IVA, principalmente relacionados à toxicidade por hipervitaminose A, (10,11) mas com bom prognóstico apesar da continuação do tratamento com ELX/TEZ/IVA, corroborando nossa crença de que a malformação congênita desconhecida do nosso paciente foi determinante para o desfecho negativo.…”

Section: Discussionunclassified

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Elexacaftor/tezacaftor/ivacaftor—real-world clinical effectiveness and safety. A single-center Portuguese study

et al. 2023

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Objective: To evaluate the effectiveness of treatment with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) and to characterize its safety profile in cystic fibrosis (CF) patients in a real-world clinical setting. Methods: This was a prospective observational study carried out in a CF referral center in Portugal involving adult CF patients who started treatment with ELX/TEZ/IVA. Clinical characteristics of the patients were collected, and effectiveness and safety data were evaluated. Results: Of the 56 patients followed in the center at the time of the study, 28 were eligible for ELX/TEZ/IVA treatment in accordance with the Portuguese National Authority for Medicines and Health Products at the time of the study. Of these, 24 met the follow-up time requirement to be included in the clinical effectiveness analysis. The mean follow-up time was 167.3 ± 96.4 days. Adverse events were generally mild and self-limited. Significant improvements in lung function, BMI, sweat chloride concentration, and number of pulmonary exacerbations were observed. No significant differences in outcomes between F508del homozygous and heterozygous patients were found. The effectiveness of this new CFTR modulator combination also applied to patients with advanced lung disease. Conclusions: Treatment with ELX/TEZ/IVA showed effective improvement in real-world clinical practice, namely in lung function, BMI, sweat chloride concentration, and number of pulmonary exacerbations, with no safety concerns.

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Section: Discussionsupporting

confidence: 79%

Section: Discussionunclassified

Elexacaftor/tezacaftor/ivacaftor—real-world clinical effectiveness and safety. A single-center Portuguese study

et al. 2023

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“…In the current era, frank micronutrient deficiencies of vitamin A and vitamin E are rarely encountered in patients with CF, presumably owing to early diagnosis and initiation of pancreatic enzymes, CF‐specific vitamin supplementation, and possibly improved absorption with HEMs. On the contrary, instances of elevated vitamin levels are now seen 12–17 . Also, once solely considered as micronutrients, our latest understanding of the role of fat‐soluble vitamins and trace minerals has evolved.…”

Section: Introductionmentioning

confidence: 99%

“…Higher retinol levels with possible toxicity may be encountered in patients treated predominantly with retinol‐based supplements, patients who have chronic kidney disease, and patients posttransplant 15,42,61‐63 . There are case reports of hypervitaminosis A in patients treated with CFTR modulator therapy, and more studies are needed to elucidate the effect of HEMs on vitamin A status in patients with CF 12‐14 …”

Section: Introductionmentioning

confidence: 99%

Update on the management of vitamins and minerals in cystic fibrosis

2022

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Advancements in respiratory and nutrition management have significantly improved the survival of patients with cystic fibrosis (CF). With the availability of several nutrition interventions such as oral/enteral nutrition supplements, enteric‐coated pancreatic enzymes, and water‐miscible CF‐specific vitamin supplements, frank vitamin deficiencies—with the exception of vitamin D—are rarely encountered in current clinical practice. Whereas they were previously considered as micronutrients, our current understanding of fat‐soluble vitamins and minerals as antioxidants, immunomodulators, and disease biomarkers has been evolving. The impact of highly effective modulators on the micronutrient status of patients with CF remains elusive. This narrative review focuses on the updates on the management of fat‐soluble vitamins and other micronutrients in CF in the current era and identifies the gaps in our knowledge.

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“…Presence of Mycobacterium abscessus infection for over 12 years, was no longer seen in culture in one PwCF after starting ETI 49 . Vitamin levels were altered, with vitamin E levels increasing 12 months after ETI in a review of 76 patients 50 and 2 reported cases of papilledema with elevated Vitamin A levels 51 . Mental status changes (mainly mental fogginess) were seen in a case series of 6 patients, all stopping the medication withing 3 months of initiation, despite attempts at altered dosing regimens 52 .…”

Section: Multisystem Effectsmentioning

confidence: 99%

Cystic fibrosis year in review 2021

Savant

2022

Preprint

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People with cystic fibrosis (CF) have an amazing outlook with the treatment availability of highly effective modulators. Unfortunately, not all PwCF are eligible for modulators leading to continued pulmonary exacerbations and advanced lung disease. Additionally, optimizing diagnosis and evaluation for CF in the newborn period continues to be an area of focus for research. This review article will work to cover articles published in 2021 with high clinical relevance related to the above topics, however due to the extensive body of research published, this review will not be comprehensive.

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Papilledema and hypervitaminosis A after elexacaftor/tezacaftor/ivacaftor for cystic fibrosis

Cited by 22 publications

References 10 publications

Elexacaftor/tezacaftor/ivacaftor—real-world clinical effectiveness and safety. A single-center Portuguese study

Elexacaftor/tezacaftor/ivacaftor—real-world clinical effectiveness and safety. A single-center Portuguese study

Update on the management of vitamins and minerals in cystic fibrosis

Cystic fibrosis year in review 2021

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