Ivacaftor drug desensitization

Patterson, Alexander; Autry, Elizabeth; Kuhn, Robert; Wurth, Mark

doi:10.1002/ppul.24319

Cited by 13 publications

(17 citation statements)

References 4 publications

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“…The majority of the included studies and case series/reports regarded LUM/IVA, totaling 47 (69%) of the 68 included. IVA, TEZ/IVA, and ELX/TEZ/IVA were the subject of 11 (16%), 7 (10%), and 4 (6%) of the included manuscripts and abstracts, respectively (one study discussed both LUM/IVA and TEZ/IVA, so was included in the totals for both modulators [28]). Eleven (16%) of the included studies and case series/reports included only pediatrics (age < 18 years old), 33 (49%) included only adults, 16 (24%) included both adult and pediatric patients, and the age groups included was not specified in 8 (12%).…”

Section: Description Of Studiesmentioning

confidence: 99%

“…Fourteen (26%) of the observational studies focused on patients with severe airflow obstruction, defined as ppFEV 1 < 40%; in 11 of these studies, patients accessed the CFTR modulator via a compassionate access program (also referred to as a "managed access", "early access', "expanded access", or "named patient" program) through the manufacturer [25,26,[29][30][31][32][33][34][35][36][37][38][39][40]. Twenty (65%) of the full manuscripts were deemed to have safety as a primary focus or outcome [27][28][29][30]32,33,[41][42][43][44][45][46][47][48][49][50][51][52][53][54].…”

Section: Description Of Studiesmentioning

confidence: 99%

See 1 more Smart Citation

Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review

Dagenais

Quon

2020

JCM

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Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies target the underlying cause of cystic fibrosis (CF), and are generally well-tolerated; however, real-world studies indicate the frequency of discontinuation and adverse events (AEs) may be higher than what was observed in clinical trials. The objectives of this systematic review were to summarize real-world AEs reported for market-available CFTR modulators (i.e., ivacaftor (IVA), lumacaftor/ivacaftor (LUM/IVA), tezacaftor/ivacaftor (TEZ/IVA), and elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA)), and to identify ways in which the pharmacist on CF healthcare teams may contribute to mitigating and managing these AEs. The MEDLINE, EMBASE, CINAHL, and Web of Science Core Collection online databases were searched from 2012 to 1 Aug 2020. Full manuscripts or conference abstracts of observational studies, case series, and case reports were eligible for inclusion. The included full manuscripts and conference abstracts comprised of 54 observational studies, 5 case series, and 9 case reports. The types of AEs reported generally aligned with what have been observed in clinical trials. LUM/IVA was associated with a higher frequency of respiratory-related AE and discontinuation in real-world studies. A signal for mental health and neurocognitive AEs was identified with all 4 CFTR modulators. A systematic approach to monitoring for AEs in people with CF on CFTR modulators in the real-world setting is necessary to help better understand potential AEs, as well as patient characteristics that may be associated with higher risk of certain AEs. Pharmacists play a key role in the safe initiation and monitoring of people with CF on CFTR modulator therapies.

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Section: Description Of Studiesmentioning

confidence: 99%

Section: Description Of Studiesmentioning

confidence: 99%

Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review

Dagenais

Quon

2020

JCM

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“…Successful desensitization occurred with dilutions of ivacaftor, increased over 10 days, followed by a transition to tezacaftor/ivacaftor. For the subsequent 6 months, he had no further drug reaction 34 …”

Section: Modulator‐specific Effectsmentioning

confidence: 96%

Cystic fibrosis year in review 2019: Section 1 CFTR modulators

Savant

McColley

2020

Pediatric Pulmonology

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During the year 2019, research and case reports/series in the field of cystic fibrosis (CF) were in abundance. To adequately address the large body of CF research published during 2019, the CF year in review will be divided into three sections. This report is the first section, focusing specifically on new research related to cystic fibrosis transmembrane conductance regulator modulator therapy. Additional sections will concentrate on pulmonary and infections research and the multisystem effects of CF. It is an exciting time to be providing care for patients and their families with CF with all the exciting new discoveries that will be shared in these reviews.

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“…The protocol developed was based on a protocol previously reported in the literature for ivacaftor. 10 Dilutions of tezacaftor/ivacaftor were prepared using crushed tablets mixed with a fixed oil, which consisted of almond oil with silica gel. The patient was instructed to take each dose with whole milk and high fat food.…”

Section: Casementioning

confidence: 99%

“…She was initiated on tezacaftor/ivacaftor after Food and Drug Administration (FDA) approval and developed a rash consistent with her previous drug rashes. She successfully completed a 10‐day desensitization protocol to ivacaftor and was able to transition to tezacaftor/ivacaftor combination therapy without report of any adverse reaction 10 . Upon initiation of elexacaftor/tezacaftor/ivacaftor, she developed a diffuse, red rash with small papules that began on her trunk and spread outwards, 7 days after initiation.…”

Section: Introductionmentioning

confidence: 99%

CFTR modulator drug desensitization: Preserving the hope of long term improvement

Leonhardt

Autry

Kuhn

et al. 2021

Pediatric Pulmonology

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The development of modulator therapy has, for the first time, allowed direct targeting of the underlying cause of cystic fibrosis (CF), the cystic fibrosis transmembrane conductance regulator (CFTR). Patients treated with CFTR modulators have improvement in lung function and decreased rates of pulmonary exacerbations. In 2019, elexacaftor/tezacaftor/ivacaftor was approved for use in the United States, opening these therapies to 90% of patients with CF. Intolerable adverse drug reactions to CFTR modulators results in discontinuation of therapy, which can be devastating to our patients. We describe our approach to two cases, not previously

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Ivacaftor drug desensitization

Cited by 13 publications

References 4 publications

Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review

Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review

Cystic fibrosis year in review 2019: Section 1 CFTR modulators

CFTR modulator drug desensitization: Preserving the hope of long term improvement

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