Tackling the increasing complexity of CF care

Sawicki, Gregory S.; Goss, Christopher H.

doi:10.1002/ppul.23244

Cited by 37 publications

(36 citation statements)

References 61 publications

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“…Because it is difficult for CF clinicians to keep all these interactions in mind, case management by pharmacists/pharmacologists for CF care is highly recommended for identification of and education concerning drug–drug interactions and other potentially troublesome drug combinations. Research evaluating comparative effectiveness of existing therapies may provide a framework for individualized, potentially simplified regimens in the midst of ongoing development and implementation of new therapeutics …”

Section: Discussionmentioning

confidence: 99%

Therapeutic challenges posed by critical drug–drug interactions in cystic fibrosis

Jordan

Noah

Henry

2016

Pediatric Pulmonology

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This review seeks to re-introduce cystic fibrosis (CF) clinicians to the pharmacology of drug-drug interactions among medications commonly used in CF and provide a framework for understanding these interactions among medications outside the scope of this discussion. We here focus on drugs impacted by the cytochrome P-450 (CYP450) enzyme system and on interactions involving antimicrobials, psychotropic medications, and cystic fibrosis transmembrane conductance regulator (CFTR) modulators. Particular attention is needed when prescribing rifampin, azole antifungals and the CFTR modulators, ivacaftor, and lumacaftor/ivacaftor, in combination with other medications. The complexities of these interactions provide a strong rationale for case management by pharmacists and pharmacologists as a routine part of CF care. Pediatr Pulmonol. 2016;51:S61-S70. © 2016 Wiley Periodicals, Inc.

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Section: Discussionmentioning

confidence: 99%

Therapeutic challenges posed by critical drug–drug interactions in cystic fibrosis

Jordan

Noah

Henry

2016

Pediatric Pulmonology

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“…These decisions often affect both the exposure and outcome of interest. This type of bias creates statistical challenges in the development of models to examine comparative effectiveness, and the issues have been highlighted over the years (37) (38),(39). A pervasive example is when using a retrospective cohort to compare clinical outcomes either between patients receiving a particular therapy versus not or pre- to post therapy, as has been done for commonly used CF therapies such as inhaled tobramycin(25), rhDNase(24), and inhaled corticosteroid therapy(23).…”

Section: Fev1 In Cf Epidemiologic Studies: Analytic Approaches Formentioning

confidence: 99%

Use of FEV1 in cystic fibrosis epidemiologic studies and clinical trials: A statistical perspective for the clinical researcher

Szczesniak

Heltshe

Stanojevic

et al. 2017

Journal of Cystic Fibrosis

106

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Background Forced expiratory volume in 1 second (FEV1) is an established marker of cystic fibrosis (CF) disease progression that is used to capture clinical course and evaluate therapeutic efficacy. The research community has established FEV1 surveillance data through a variety of observational data sources such as patient registries, and there is a growing pipeline of new CF therapies demonstrated to be efficacious in clinical trials by establishing improvements in FEV1. Results In this review, we summarize from a statistical perspective the clinical relevance of FEV1 based on its association with morbidity and mortality in CF, its role in epidemiologic studies of disease progression and comparative effectiveness, and its utility in clinical trials. In addition, we identify opportunities to advance epidemiologic research and the clinical development pipeline through further statistical considerations. Conclusions Our understanding of CF disease course, therapeutics, and clinical care has evolved immensely in the past decades, in large part due to the thoughtful application of rigorous research methods and meaningful clinical endpoints such as FEV1. A continued commitment to conduct research that minimizes the potential for bias, maximizes the limited patient population, and harmonizes approaches to FEV1 analysis while maintaining clinical relevance, will facilitate further opportunities to advance CF care.

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“…However, recent years have also seen an increase in disease complexity [3] with newer, more resistant genetic variants [4] often emerging. Some of these have been covered very well in several excellent reviews [3, 5–7]. One common theme that gets less than deserved attention is the interplay of immediate environmental factors in shaping the course of disease progression.…”

Section: Introductionmentioning

confidence: 99%

Cystic fibrosis lung environment and Pseudomonas aeruginosa infection

et al. 2016

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BackgroundThe airways of patients with cystic fibrosis (CF) are highly complex, subject to various environmental conditions as well as a distinct microbiota. Pseudomonas aeruginosa is recognized as one of the most important pulmonary pathogens and the predominant cause of morbidity and mortality in CF. A multifarious interplay between the host, pathogens, microbiota, and the environment shapes the course of the disease. There have been several excellent reviews detailing CF pathology, Pseudomonas and the role of environment in CF but only a few reviews connect these entities with regards to influence on the overall course of the disease. A holistic understanding of contributing factors is pertinent to inform new research and therapeutics.DiscussionIn this article, we discuss the deterministic alterations in lung physiology as a result of CF. We also revisit the impact of those changes on the microbiota, with special emphasis on P. aeruginosa and the influence of other non-genetic factors on CF. Substantial past and current research on various genetic and non-genetic aspects of cystic fibrosis has been reviewed to assess the effect of different factors on CF pulmonary infection. A thorough review of contributing factors in CF and the alterations in lung physiology indicate that CF lung infection is multi-factorial with no isolated cause that should be solely targeted to control disease progression. A combinatorial approach may be required to ensure better disease outcomes.ConclusionCF lung infection is a complex disease and requires a broad multidisciplinary approach to improve CF disease outcomes. A holistic understanding of the underlying mechanisms and non-genetic contributing factors in CF is central to development of new and targeted therapeutic strategies.

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Tackling the increasing complexity of CF care

Cited by 37 publications

References 61 publications

Therapeutic challenges posed by critical drug–drug interactions in cystic fibrosis

Therapeutic challenges posed by critical drug–drug interactions in cystic fibrosis

Use of FEV1 in cystic fibrosis epidemiologic studies and clinical trials: A statistical perspective for the clinical researcher

Cystic fibrosis lung environment and Pseudomonas aeruginosa infection

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