Search Results - "Hamblett, N."

Accelerated Approval or Risk Reduction? How Response Biomarkers Advance Therapeutics through Clinical Trials in Cystic Fibrosis by Mayer-Hamblett, N., VanDevanter, D.R.

“…Progress in the development of new therapies for cystic fibrosis (CF) has benefited from therapeutically responsive biomarkers to streamline drug development…”
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Heterogeneity and Disparities in the Use of Exception Scores in Pediatric Liver Allocation by Hsu, E. K., Shaffer, M., Bradford, M., Mayer‐Hamblett, N., Horslen, S.

“…Physicians apply for Model for End‐Stage Liver Disease/Pediatric End‐Stage Liver Disease exception points on a case‐by‐case basis to improve an individual…”
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KB001-A, a novel anti-inflammatory, found to be safe and well-tolerated in cystic fibrosis patients infected with Pseudomonas aeruginosa by Jain, Raksha, Beckett, V.V., Konstan, M.W., Accurso, F.J., Burns, J.L., Mayer-Hamblett, N., Milla, Carlos, VanDevanter, D.R., Chmiel, J.F.

“…Chronic Pseudomonas aeruginosa (Pa) airways infection, exuberant local inflammation, and progressive lung function loss are hallmarks of cystic fibrosis (CF)…”
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Important steps in the journey to highly effective CFTR modulator access for people with CF by VanDevanter, D.R, Mayer-Hamblett, N

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Heterogeneity of CFTR modulator-induced sweat chloride concentrations in people with cystic fibrosis by Zemanick, E.T., Emerman, I., McCreary, M., Mayer-Hamblett, N., Warden, M.N., Odem-Davis, K., VanDevanter, D.R., Ren, C.L., Young, J., Konstan, M.W.

“…•Using data from the CHEC-SC study of modulator-induced sweat chloride (SC) concentrations, we describe the proportion of individuals with post-modulator SC…”
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Willingness of people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor (ETI) to participate in randomized modulator and inhaled antimicrobial clinical trials by VanDevanter, D.R., Zemanick, E.T., Konstan, M.W., Ren, C.L., Odem-Davis, K., Emerman, I., Young, J., Mayer-Hamblett, N.

“…•Willingness to enroll in clinical trials was assessed for those on CFTR modulators.•Willingness to enroll was associated with proposed trial length and…”
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Characterizing CFTR modulated sweat chloride response across the cf population: Initial results from the CHEC-SC study by Mayer-Hamblett, N, Zemanick, ET, Odem-Davis, K, VanDevanter, D, Warden, M, Rowe, SM, Young, J, Konstan, MW, for-the-CHEC-SC-Study-Group

“…•Modulator-induced sweat chloride (SC) responses across the CF population were consistent with those reported from prior clinical studies.•Variation in SC…”
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Another step in the journey: From CFTR mutation to sweat chloride concentration to survival by VanDevanter, D.R., Mayer-Hamblett, N.

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Building global development strategies for cf therapeutics during a transitional cftr modulator era by Mayer-Hamblett, N., van Koningsbruggen-Rietschel, S., Nichols, D.P., VanDevanter, D.R., Davies, J.C., Lee, T., Durmowicz, A.G., Ratjen, F., Konstan, M.W., Pearson, K., Bell, S.C., Clancy, J.P., Taylor-Cousar, J.L., De Boeck, K., Donaldson, S.H., Downey, D.G., Flume, P.A., Drevinek, P., Goss, C.H., Fajac, I., Magaret, A.S., Quon, B.S., Singleton, S.M., VanDalfsen, J.M., Retsch-Bogart, G.Z.

“…•Advancement of the CF therapeutics pipeline remains an important goal.•Availability of CFTR modulators impacts future CF clinical development.•Global…”
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In statistics we trust: Towards the careful derivation and interpretation of meaningful survival estimates in cystic fibrosis by Mayer-Hamblett, N., Polineni, D., Heltshe, S.L.

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Risk of hemoptysis in cystic fibrosis clinical trials: A retrospective cohort study by Thompson, V, Mayer-Hamblett, N, Kloster, M, Bilton, D, Flume, P.A

“…Abstract Background Cystic fibrosis (CF) is characterized by airway infection and inflammation resulting in respiratory complications including hemoptysis. The…”
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65 Physician assessment of blinded adverse events in RCTs in cystic fibrosis by Wessels, M.E, Mayer-Hamblett, N, Ramsey, B.W, Heltshe, S, Baines, A, Goss, C.H

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Corrigendum to “Risk of hemoptysis in cystic fibrosis clinical trials: A retrospective cohort study” [J Cyst Fibros (2015) 632–638] by Thompson, V, Mayer-Hamblett, N, Kloster, M, Bilton, D, Flume, P.A

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Significant Microbiological Effect of Inhaled Tobramycin in Young Children with Cystic Fibrosis by Gibson, Ronald L, Emerson, Julia, McNamara, Sharon, Burns, Jane L, Rosenfeld, Margaret, Yunker, Ann, Hamblett, Nicole, Accurso, Frank, Dovey, Mark, Hiatt, Peter, Konstan, Michael W, Moss, Richard, Retsch-Bogart, George, Wagener, Jeffrey, Waltz, David, Wilmott, Robert, Zeitlin, Pamela L, Ramsey, Bonnie

“…We conducted a double-blind, placebo-controlled, multicenter, randomized trial to test the hypothesis that 300 mg of tobramycin solution for inhalation…”
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Association between Stenotrophomonas maltophilia and lung function in cystic fibrosis by Goss, C H, Mayer-Hamblett, N, Aitken, M L, Rubenfeld, G D, Ramsey, B W

“…Background:Stenotrophomonas maltophilia (SM) is a Gram-negative non-fermenting bacteria cultured from the sputum of patients with cystic fibrosis (CF). To…”
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54 Relationship between rate of percent predicted FEV1 (ppFEV1 ) decline and baseline and acute change in ppFEV1 in patients (pts) with cystic fibrosis (CF) treated with lumacaftor/ivacaftor (LUM/IVA) by Konstan, M.W, McKone, E.F, Moss, R.B, Lubarsky, B, Rubin, J, Millar, S.J, Pasta, D.J, Mayer-Hamblett, N, Goss, C.H, Morgan, W, Sawicki, G.S

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Inflammatory and Microbiologic Markers in Induced Sputum after Intravenous Antibiotics in Cystic Fibrosis by Ordonez, Claudia L, Henig, Noreen R, Mayer-Hamblett, Nicole, Accurso, Frank J, Burns, Jane L, Chmiel, James F, Daines, Cori L, Gibson, Ronald L, McNamara, Sharon, Retsch-Bogart, George Z, Zeitlin, Pamela L, Aitken, Moira L

“…Induced sputum has been used to study airway inflammation. We sought to determine whether markers of infection and inflammation in induced sputum were a useful…”
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Rationale and design of a randomized trial of home electronic symptom and lung function monitoring to detect cystic fibrosis pulmonary exacerbations: The early intervention in cystic fibrosis exacerbation (eICE) trial by Lechtzin, N, West, N, Allgood, S, Wilhelm, E, Khan, U, Mayer-Hamblett, N, Aitken, M.L, Ramsey, B.W, Boyle, M.P, Mogayzel, P.J, Goss, C.H

“…Abstract Background Acute pulmonary exacerbations are central events in the lives of individuals with cystic fibrosis (CF). Pulmonary exacerbations lead to…”
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WS02.1 Efficacy of a protocol for eradication of newly acquired MRSA: Results of the STAR-too trial by Goss, C.H, Thompson, V, Popowitch, E, Howe, D.L, Baines, A, Mayer-Hamblett, N, Jill, V, Muhlebach, M.S

“…Objectives The ST aph A ureus R esistance – t reat o r o bserve trial (STAR-too) evaluates microbiologic effectiveness of an eradication protocol (Rx) compared…”
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112 Analysis of long-term use of liposomal amikacin for inhalation (LAI) in patients with cystic fibrosis (CF) who have chronic infection from Pseudomonas aeruginosa by Bilton, D, Pressler, T, Fajac, I, Clancy, J.P, Minic, P, Cipolli, M, Galeva, I, Solé, A, Dupont, L.J, Mayer-Hamblett, N, Torchio, S, McGinnis, J.P, Eagle, G, Konstan, M

“…Objective Interim analysis to evaluate safety, tolerability, and efficacy of LAI, a novel amikacin formulation, in CF patients with chronic P aeruginosa…”
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