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| drugs_cystic_fibrosis [2026/08/03 10:29] – Scott Larson | drugs_cystic_fibrosis [2026/08/03 11:13] (current) – [References] Scott Larson | ||
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| ====== Drugs for Cystic Fibrosis ====== | ====== Drugs for Cystic Fibrosis ====== | ||
| - | === Trikafta=== | + | * **Trikafta** (elexacaftor/ |
| - | elexacaftor/ | + | * **Kalydeco** (ivacaftor) |
| - | + | * **Orkambi** (lumacaftor/ | |
| - | * transmembrane conductance regulator potentiator | + | * **Symdeko** (tezacaftor/ |
| - | * Approved for ages 2 and older | + | * **Alyftrek** (vanzacaftor/tezacaftor/deutivacaftor) |
| - | * [[https://www-bmj-com.proxy.lib.uiowa.edu/ | + | * Outside North America, Trikafta is marketed as Kaftrio, and Symdeko as Symkevi |
| - | + | * this class of drugs regular transmembrane conductance | |
| - | ==== Cataracts with Lumacaftor/ | + | |
| + | ==== Cataracts with Lumacaftor/ | ||
| * Cataracts were found in juvenile rats in pre-clinical toxicology studies | * Cataracts were found in juvenile rats in pre-clinical toxicology studies | ||
| * Cataracts have been discovered in a few children taking this drug. The concomitant use of steroids or other pre-existing coniditions could not be excluded. | * Cataracts have been discovered in a few children taking this drug. The concomitant use of steroids or other pre-existing coniditions could not be excluded. | ||
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| === Cataracts / Lens Opacities === | === Cataracts / Lens Opacities === | ||
| - | * Non-congenital lens opacities have been reported in pediatric patients on ivacaftor-containing regimens. Confounders (corticosteroid use, radiation exposure) were present in some cases, but a drug-attributable risk cannot be excluded.*[1] | + | * Non-congenital lens opacities have been reported in pediatric patients on ivacaftor-containing regimens. Confounders (corticosteroid use, radiation exposure) were present in some cases, but a drug-attributable risk cannot be excluded.[1] |
| - | * Signal originates from juvenile animal toxicology: cataracts occurred in rats dosed from postnatal day 7 through 35 at ivacaftor >=10 mg/kg/day (0.21x MRHD by systemic exposure); not seen in older animals.*[1] | + | * Signal originates from juvenile animal toxicology: cataracts occurred in rats dosed from postnatal day 7 through 35 at ivacaftor >=10 mg/kg/day (0.21x MRHD by systemic exposure); not seen in older animals.[1] |
| - | * In clinical trials, cataracts are classified as an **uncommon** adverse event across all CFTR modulators, including ETI, and were not observed as a treatment effect in human trials.*[2][3] | + | * In clinical trials, cataracts are classified as an **uncommon** adverse event across all CFTR modulators, including ETI, and were not observed as a treatment effect in human trials.[2][3] |
| === Intracranial Hypertension === | === Intracranial Hypertension === | ||
| - | * Reported postmarketing; | + | * Reported postmarketing; |
| - | * **Patients with elevated vitamin A levels may be at increased risk** — relevant given routine fat-soluble vitamin supplementation in CF.*[1] | + | * **Patients with elevated vitamin A levels may be at increased risk** — relevant given routine fat-soluble vitamin supplementation in CF.[1] |
| - | * If unusual headache or visual disturbance occurs and IH is suspected, **interrupt Trikafta and refer for prompt medical evaluation**; | + | * If unusual headache or visual disturbance occurs and IH is suspected, **interrupt Trikafta and refer for prompt medical evaluation**; |
| === Other Reported Ocular Symptoms === | === Other Reported Ocular Symptoms === | ||
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| ==== Practical Monitoring Approach ==== | ==== Practical Monitoring Approach ==== | ||
| + | <WRAP round box 85%> | ||
| ^ Element ^ Recommendation ^ | ^ Element ^ Recommendation ^ | ||
| | Baseline eye exam | Recommended in pediatric patients before/at initiation of ETI | | | Baseline eye exam | Recommended in pediatric patients before/at initiation of ETI | | ||
| - | | Follow-up exams | Follow-up ophthalmologic examinations recommended in pediatric patients; | + | | Follow-up exams | Follow-up ophthalmologic examinations recommended in pediatric patients; |
| | Adults | No routine screening exam mandated by label; evaluate symptom-driven| | | Adults | No routine screening exam mandated by label; evaluate symptom-driven| | ||
| | New headache or visual disturbance (any age) | Interrupt drug; urgent evaluation including fundoscopy for papilledema; | | New headache or visual disturbance (any age) | Interrupt drug; urgent evaluation including fundoscopy for papilledema; | ||
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| The recommended exam should include **slit-lamp assessment for lens opacities and fundoscopy**. The label does not specify a fixed interval beyond " | The recommended exam should include **slit-lamp assessment for lens opacities and fundoscopy**. The label does not specify a fixed interval beyond " | ||
| + | </ | ||
| =====References===== | =====References===== | ||
| - | 1. Trikafta. Food and Drug Administration. Updated date: 2026-06-22. | + | - [[https:// |
| - | 2. Cystic Fibrosis. Grasemann H, Ratjen F. The New England Journal of Medicine. 2023; | + | - [[https:// |
| - | 3. Cystic Fibrosis. Ong T, Ramsey BW. JAMA. 2023; | + | - [[https://pubmed.ncbi.nlm.nih.gov/ |
| - | 4. Heterogeneity in Reported Side Effects Following Initiation of Elexacaftor‐Tezacaftor‐Ivacaftor: | + | - [[https://pubmed.ncbi.nlm.nih.gov/ |
| + | - [[https://pmc.ncbi.nlm.nih.gov/articles/ | ||
| {{tag> | {{tag> | ||