We Need to Widen Inclusion Criteria for Cystic Fibrosis Clinical Trials
As readers of my column, you know that I write about the journey of life and the emotions of living with cystic fibrosis (CF). This week, I want to address a topic that makes me sad and frustrated. I’ve made it my mission to shine a light on the current clinical trial inclusion criteria — specifically minimum lung function.
I’ve been excluded from almost all trials involving investigative treatments for CF, due to having lung function below the 40 percent forced expiratory volume in one second (FEV1) inclusion limit. It’s frustrating that I’m ineligible for participation in clinical trials because of this one criterion. I know that I am not alone — many adult patients with low FEV1 have a good quality of life and live with far greater levels of general health and physical fitness than our FEV1 would suggest.
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I may never benefit from new medications in the future because my health is deteriorating, and I may require a lung transplant before FDA approval has been granted for these medications. After transplant, my new lungs won’t have CF, and those medicines will not be as vital to my care.
But I’m choosing not to dwell on these aspects. Instead, I will turn my frustration into action.
I wrote a column published by MedPage Today that describes this situation. In it, I suggested alternative selection criteria and methods. I hope to reach those responsible for determining clinical trial criteria, specifically the 40 percent FEV1 inclusion limit. I am deeply motivated and passionate about this, and I hope my message will reach those who need to hear the perspective of a person with cystic fibrosis.
Thank you for reading and sharing.
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Note: Cystic Fibrosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Cystic Fibrosis News Today, or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to cystic fibrosis.
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Tim Blowfield
Even with lung transplantation you will still have CF but not in your new lungs. The mutant CFTR is in every cell of a CF person's body even those cells we rarely suspect are affected. As CF people get older the effects of the faulty CFTR may show up in surprising ways - my wife is 76 - she has CF, cardiomyopathy, Sjogren's Syndrome, Multiple endocrine disorders (hyperparathyroidism, Adrenal failure, there is a question about her pituitary though no one is willing to investigate that). How do these relate to the CF that was there first? How do we get Drs to treat her as a whole rather than just the part they specialised in. The cardiologist sees only the heart and not the effect of the CF, Sjogren's nor even the endocrine glands on it. Endocrine specialists may just see the hyperparathyroidism and ignore the Adrenals. A rheumatologist put her in the box of polymyalgia rheumatica, a misdiagnosis of the Sjogren's. Even pulmonologist CF Drs too often ignore the rest of the body because the lungs are so important.