The Role of Dornase Alfa in the Treatment of Cystic Fibrosis

Cramer, Gena W.; Bosso, John A.; Maldonado, Wanda T; Guévremont, Chantal

doi:10.1177/106002809603000614

Cited by 13 publications

(7 citation statements)

References 13 publications

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“…As reports of the G‐CSF priming of neutrophils to form NETs are numerous, anti‐G‐CSF may be useful to prevent NETs in patients. This is likely to be more successful than the administration of an agent such as DNase to disrupt other NET components, which is currently in use for cystic fibrosis . Thus, strategies disrupting G‐CSF signalling require further investigation in vivo , especially with regard to effects on non‐tumour‐induced granulopoiesis that is required to fight infection.…”

Section: Neutrophils and G‐csf As Therapeutic Targetsmentioning

confidence: 99%

Neutrophils, G‐CSF and their contribution to breast cancer metastasis

2017

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Evidence is mounting for a role for neutrophils in breast cancer progression to metastasis. However, the role of G‐CSF in neutrophil biology in a cancer setting remains to be defined. Herein we discuss the most recent clinical and experimental evidence for neutrophils and G‐CSF in the promotion of metastasis, demonstrating a potential mechanistic link between them. Understanding this link is imperative both for the development of diagnostic tests and for therapies targeting neutrophils to improve the treatment of breast cancer patients with or at risk of developing metastatic disease. As a high neutrophil‐to‐lymphocyte ratio in patients predicts poor outcome, while mild neutropenia predicts an improved outcome, we urge caution in the use of G‐CSF in neutrophil recovery following chemotherapy as there is increasing evidence in preclinical models that G‐CSF can promote metastasis.

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Section: Neutrophils and G‐csf As Therapeutic Targetsmentioning

confidence: 99%

Neutrophils, G‐CSF and their contribution to breast cancer metastasis

2017

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“…19 Its safety was well established and its short term efficacy seemed reasonable, but its long term efficacy and specific indications for its use in patients with cystic fibrosis were not apparent 2021…”

Section: Discussionmentioning

confidence: 99%

Long term effects of aerosolised rhDNase on pulmonary disease progression in patients with cystic fibrosis

Milla

1998

Thorax

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Background-After multiple studies, including clinical trials, suggested some mild clinical benefits from the use of rhDNase by patients with cystic fibrosis, a widespread acceptance of the drug has followed. However, long term eVects, specifically on lung disease progression, have not been demonstrated. Experience with the use of this drug in a single cystic fibrosis centre is presented and compared with the trends seen in the patient population of the centre before the introduction of the drug. Methods-Patients with cystic fibrosis routinely followed at the University of Minnesota Cystic Fibrosis Center and prescribed rhDNase for at least two years were included in this retrospective study. Data on spirometric parameters (FEV 1 and FEV 1 /FVC), allometric index, and admissions to hospital were retrieved from the centre's database for the two years preceding the prescription of rhDNase and the two years that followed. Trends in pulmonary function and allometric index were analysed by mixed linear modelling, and hospital admission rates for both periods were calculated and compared. Results-One hundred and ninety patients met the inclusion criteria for the study. In the two years preceding the prescription of rhDNase the trends noted were those of a mild decline in FEV 1 , a stable FEV 1 /FVC, and a mild improvement in allometric index. In the two years that followed the prescription of rhDNase a mild decline in all these parameters occurred which was a significant change from the previous period (all p <0.009). There was no diVerence between females and males in the trends experienced after the start of rhDNase. By logistic regression analysis only the presence of malnutrition at the time of prescription was associated with a positive trend after the introduction of rhDNase. No significant change in the hospital admission rates occurred, with rates of 0.52 (0.16) and 0.56 (0.21) admissions/patient/year for the periods before and after the prescription of rhDNase, respectively. Conclusions-The introduction of rhDNase to the regimen of patients with cystic fibrosis cared for at this centre has not been followed by a positive trend in lung function and nutritional parameters. There are some diVerences between this patient population and those who participated in previous studies which may help to explain the contrasting findings of this study. However, it is also possible that factors other than mucus clearance need to be improved to achieve a favourable response in disease progression. Patients on this treatment should be followed closely and the benefit judged on an individual basis. More studies are needed to define better the specific indications and use of this form of treatment.

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“…When dornase-alfa was compared to hypertonic saline, it was found to result in a mean improvement in FEV1 of 8% at a mean additional cost of £1 409 over a 12-week period. In addition, using alternate-day dornase-alfa was not a cost-effective alternative in comparison with daily treatment for the same period 20. In general, clinical trials in CF have shown improvements in lung function 21.…”

Section: Practical Examples Of the Usefulness Of Pharmacoeconomic Anamentioning

confidence: 98%

“…It is considered an effective mucolytic and is indicated in conjunction with standard therapies for cystic fibrosis (CF). Clinical trials have indicated a small improvement in forced expiratory volume in 1 second (FEV1) and forced vital capacity (FVC), but whether quality of life is affected in a meaningful and measurable way is yet to be clarified 18–20. When dornase-alfa was compared to hypertonic saline, it was found to result in a mean improvement in FEV1 of 8% at a mean additional cost of £1 409 over a 12-week period.…”

Section: Practical Examples Of the Usefulness Of Pharmacoeconomic Anamentioning

confidence: 99%

Pharmacoeconomic Analysis in Saudi Arabia: An Overdue Agenda Item for Action

Al-Jazairi

Al-Qadheeb

Ajlan

2011

Annals of Saudi Medicine

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Pharmacoeconomics is a branch of health economics related to the most economical and efficient use of pharmaceuticals. Pharmacoeconomic research identifies, measures and compares the costs and outcomes (clinical, economic and humanistic) of pharmaceutical products and services. Pharmacoeconomic evaluation can play a significant role in the efficient allocation of resources in healthcare systems with constrained budgets. Countries are trying to control the rising costs of health care in their aging population. They are all asking the same question: Is the new drug good value for money; and if so, what is the society willing to pay for it? This article reviews the importance of, and the need for, adaptation of pharmacoeconomic analysis to the conditions in Saudi Arabia. It will shed some light on the important steps for converting the concept into practice, including the need for identifying the willing-to-pay (WTP) or the threshold cutoff, the existence of a real cost for each utility, the nonexistence of an pharmacoeconomic advisory forum, pharmaceutical budget allocation, and the impact of pharmaceutical marketing. It will also provide recommendations for easing any challenges that might jeopardize the conduct of such analysis in Saudi Arabia.

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The Role of Dornase Alfa in the Treatment of Cystic Fibrosis

Cited by 13 publications

References 13 publications

Neutrophils, G‐CSF and their contribution to breast cancer metastasis

Neutrophils, G‐CSF and their contribution to breast cancer metastasis

Long term effects of aerosolised rhDNase on pulmonary disease progression in patients with cystic fibrosis

Pharmacoeconomic Analysis in Saudi Arabia: An Overdue Agenda Item for Action

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