Impact of Farnesylation Inhibitors on Survival in Hutchinson-Gilford Progeria Syndrome
Hutchinson-Gilford progeria syndrome is an ultrarare segmental premature aging disease resulting in early death from heart attack or stroke. There is no approved treatment, but starting in 2007, several recent single-arm clinical trials administered inhibitors of protein farnesylation aimed at reduc...
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| Published in | Circulation (New York, N.Y.) Vol. 130; no. 1; pp. 27 - 34 |
|---|---|
| Main Authors | , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
Hagerstown, MD
Lippincott Williams & Wilkins
01.07.2014
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| Subjects | |
| Online Access | Get full text |
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| Abstract | Hutchinson-Gilford progeria syndrome is an ultrarare segmental premature aging disease resulting in early death from heart attack or stroke. There is no approved treatment, but starting in 2007, several recent single-arm clinical trials administered inhibitors of protein farnesylation aimed at reducing toxicity of the disease-producing protein progerin. No study assessed whether treatments influence patient survival. The key elements necessary for this analysis are a robust natural history of survival and comparison with a sufficiently large patient population that has been treated for a sufficient time period with disease-targeting medications.
We generated Kaplan-Meier survival analyses for the largest untreated Hutchinson-Gilford progeria syndrome cohort to date. Mean survival was 14.6 years. Comparing survival for treated versus age- and sex-matched untreated cohorts, hazard ratio was 0.13 (95% confidence interval, 0.04-0.37; P<0.001) with median follow-up of 5.3 years from time of treatment initiation. There were 21 of 43 deaths in untreated versus 5 of 43 deaths among treated subjects. Treatment increased mean survival by 1.6 years.
This study provides a robust untreated disease survival profile that can be used for comparisons now and in the future to assess changes in survival with treatments for Hutchinson-Gilford progeria syndrome. The current comparisons estimating increased survival with protein farnesylation inhibitors provide the first evidence of treatments influencing survival for this fatal disease.
http://www.clinicaltrials.gov. Unique Indentifiers: NCT00425607, NCT00879034, and NCT00916747. |
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| AbstractList | Hutchinson-Gilford progeria syndrome is an ultrarare segmental premature aging disease resulting in early death from heart attack or stroke. There is no approved treatment, but starting in 2007, several recent single-arm clinical trials administered inhibitors of protein farnesylation aimed at reducing toxicity of the disease-producing protein progerin. No study assessed whether treatments influence patient survival. The key elements necessary for this analysis are a robust natural history of survival and comparison with a sufficiently large patient population that has been treated for a sufficient time period with disease-targeting medications.BACKGROUNDHutchinson-Gilford progeria syndrome is an ultrarare segmental premature aging disease resulting in early death from heart attack or stroke. There is no approved treatment, but starting in 2007, several recent single-arm clinical trials administered inhibitors of protein farnesylation aimed at reducing toxicity of the disease-producing protein progerin. No study assessed whether treatments influence patient survival. The key elements necessary for this analysis are a robust natural history of survival and comparison with a sufficiently large patient population that has been treated for a sufficient time period with disease-targeting medications.We generated Kaplan-Meier survival analyses for the largest untreated Hutchinson-Gilford progeria syndrome cohort to date. Mean survival was 14.6 years. Comparing survival for treated versus age- and sex-matched untreated cohorts, hazard ratio was 0.13 (95% confidence interval, 0.04-0.37; P<0.001) with median follow-up of 5.3 years from time of treatment initiation. There were 21 of 43 deaths in untreated versus 5 of 43 deaths among treated subjects. Treatment increased mean survival by 1.6 years.METHODS AND RESULTSWe generated Kaplan-Meier survival analyses for the largest untreated Hutchinson-Gilford progeria syndrome cohort to date. Mean survival was 14.6 years. Comparing survival for treated versus age- and sex-matched untreated cohorts, hazard ratio was 0.13 (95% confidence interval, 0.04-0.37; P<0.001) with median follow-up of 5.3 years from time of treatment initiation. There were 21 of 43 deaths in untreated versus 5 of 43 deaths among treated subjects. Treatment increased mean survival by 1.6 years.This study provides a robust untreated disease survival profile that can be used for comparisons now and in the future to assess changes in survival with treatments for Hutchinson-Gilford progeria syndrome. The current comparisons estimating increased survival with protein farnesylation inhibitors provide the first evidence of treatments influencing survival for this fatal disease.CONCLUSIONSThis study provides a robust untreated disease survival profile that can be used for comparisons now and in the future to assess changes in survival with treatments for Hutchinson-Gilford progeria syndrome. The current comparisons estimating increased survival with protein farnesylation inhibitors provide the first evidence of treatments influencing survival for this fatal disease.http://www.clinicaltrials.gov. Unique Indentifiers: NCT00425607, NCT00879034, and NCT00916747.CLINICAL TRIAL REGISTRATION URLhttp://www.clinicaltrials.gov. Unique Indentifiers: NCT00425607, NCT00879034, and NCT00916747. Hutchinson-Gilford progeria syndrome is an ultrarare segmental premature aging disease resulting in early death from heart attack or stroke. There is no approved treatment, but starting in 2007, several recent single-arm clinical trials administered inhibitors of protein farnesylation aimed at reducing toxicity of the disease-producing protein progerin. No study assessed whether treatments influence patient survival. The key elements necessary for this analysis are a robust natural history of survival and comparison with a sufficiently large patient population that has been treated for a sufficient time period with disease-targeting medications. We generated Kaplan-Meier survival analyses for the largest untreated Hutchinson-Gilford progeria syndrome cohort to date. Mean survival was 14.6 years. Comparing survival for treated versus age- and sex-matched untreated cohorts, hazard ratio was 0.13 (95% confidence interval, 0.04-0.37; P<0.001) with median follow-up of 5.3 years from time of treatment initiation. There were 21 of 43 deaths in untreated versus 5 of 43 deaths among treated subjects. Treatment increased mean survival by 1.6 years. This study provides a robust untreated disease survival profile that can be used for comparisons now and in the future to assess changes in survival with treatments for Hutchinson-Gilford progeria syndrome. The current comparisons estimating increased survival with protein farnesylation inhibitors provide the first evidence of treatments influencing survival for this fatal disease. http://www.clinicaltrials.gov. Unique Indentifiers: NCT00425607, NCT00879034, and NCT00916747. |
| Author | Massaro, Joe Brown, W. Ted D’Agostino, Ralph B. Brazier, Joan Kleinman, Monica E. Kieran, Mark W. Gordon, Leslie B. Campbell, Susan E. |
| AuthorAffiliation | 1 Department of Pediatrics, Hasbro Children's Hospital and Warren Alpert Medical School of Brown University, Providence, RI 3 Department of Mathematics and Statistics, Boston University, Harvard Clinical Research Institute, Boston, MA 4 Center for Gerontology and Health Care Research, Brown University, Providence, RI 2 Boston Children's Hospital and Harvard Medical School, Boston, MA 6 Division of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 5 Department of Genetics, New York State Institute for Basic Research, Staten Island, NY |
| AuthorAffiliation_xml | – name: 6 Division of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA – name: 3 Department of Mathematics and Statistics, Boston University, Harvard Clinical Research Institute, Boston, MA – name: 2 Boston Children's Hospital and Harvard Medical School, Boston, MA – name: 4 Center for Gerontology and Health Care Research, Brown University, Providence, RI – name: 1 Department of Pediatrics, Hasbro Children's Hospital and Warren Alpert Medical School of Brown University, Providence, RI – name: 5 Department of Genetics, New York State Institute for Basic Research, Staten Island, NY |
| Author_xml | – sequence: 1 givenname: Leslie B. surname: Gordon fullname: Gordon, Leslie B. organization: From the Department of Pediatrics, Hasbro Children’s Hospital and Warren Alpert Medical School of Brown University, Providence, RI (L.B.G.); Department of Anesthesia, Division of Critical Care Medicine, Boston Children’s Hospital and Harvard Medical School, Boston, MA (L.B.G., M.E.K.); Department of Mathematics and Statistics, Boston University, Harvard Clinical Research Institute, Boston, MA (J.M., R.B.D.); Center for Gerontology and Health Care Research, Brown University, Providence, RI (S.E.C., J – sequence: 2 givenname: Joe surname: Massaro fullname: Massaro, Joe organization: From the Department of Pediatrics, Hasbro Children’s Hospital and Warren Alpert Medical School of Brown University, Providence, RI (L.B.G.); Department of Anesthesia, Division of Critical Care Medicine, Boston Children’s Hospital and Harvard Medical School, Boston, MA (L.B.G., M.E.K.); Department of Mathematics and Statistics, Boston University, Harvard Clinical Research Institute, Boston, MA (J.M., R.B.D.); Center for Gerontology and Health Care Research, Brown University, Providence, RI (S.E.C., J – sequence: 3 givenname: Ralph B. surname: D’Agostino fullname: D’Agostino, Ralph B. organization: From the Department of Pediatrics, Hasbro Children’s Hospital and Warren Alpert Medical School of Brown University, Providence, RI (L.B.G.); Department of Anesthesia, Division of Critical Care Medicine, Boston Children’s Hospital and Harvard Medical School, Boston, MA (L.B.G., M.E.K.); Department of Mathematics and Statistics, Boston University, Harvard Clinical Research Institute, Boston, MA (J.M., R.B.D.); Center for Gerontology and Health Care Research, Brown University, Providence, RI (S.E.C., J – sequence: 4 givenname: Susan E. surname: Campbell fullname: Campbell, Susan E. organization: From the Department of Pediatrics, Hasbro Children’s Hospital and Warren Alpert Medical School of Brown University, Providence, RI (L.B.G.); Department of Anesthesia, Division of Critical Care Medicine, Boston Children’s Hospital and Harvard Medical School, Boston, MA (L.B.G., M.E.K.); Department of Mathematics and Statistics, Boston University, Harvard Clinical Research Institute, Boston, MA (J.M., R.B.D.); Center for Gerontology and Health Care Research, Brown University, Providence, RI (S.E.C., J – sequence: 5 givenname: Joan surname: Brazier fullname: Brazier, Joan organization: From the Department of Pediatrics, Hasbro Children’s Hospital and Warren Alpert Medical School of Brown University, Providence, RI (L.B.G.); Department of Anesthesia, Division of Critical Care Medicine, Boston Children’s Hospital and Harvard Medical School, Boston, MA (L.B.G., M.E.K.); Department of Mathematics and Statistics, Boston University, Harvard Clinical Research Institute, Boston, MA (J.M., R.B.D.); Center for Gerontology and Health Care Research, Brown University, Providence, RI (S.E.C., J – sequence: 6 givenname: W. Ted surname: Brown fullname: Brown, W. Ted organization: From the Department of Pediatrics, Hasbro Children’s Hospital and Warren Alpert Medical School of Brown University, Providence, RI (L.B.G.); Department of Anesthesia, Division of Critical Care Medicine, Boston Children’s Hospital and Harvard Medical School, Boston, MA (L.B.G., M.E.K.); Department of Mathematics and Statistics, Boston University, Harvard Clinical Research Institute, Boston, MA (J.M., R.B.D.); Center for Gerontology and Health Care Research, Brown University, Providence, RI (S.E.C., J – sequence: 7 givenname: Monica E. surname: Kleinman fullname: Kleinman, Monica E. organization: From the Department of Pediatrics, Hasbro Children’s Hospital and Warren Alpert Medical School of Brown University, Providence, RI (L.B.G.); Department of Anesthesia, Division of Critical Care Medicine, Boston Children’s Hospital and Harvard Medical School, Boston, MA (L.B.G., M.E.K.); Department of Mathematics and Statistics, Boston University, Harvard Clinical Research Institute, Boston, MA (J.M., R.B.D.); Center for Gerontology and Health Care Research, Brown University, Providence, RI (S.E.C., J – sequence: 8 givenname: Mark W. surname: Kieran fullname: Kieran, Mark W. organization: From the Department of Pediatrics, Hasbro Children’s Hospital and Warren Alpert Medical School of Brown University, Providence, RI (L.B.G.); Department of Anesthesia, Division of Critical Care Medicine, Boston Children’s Hospital and Harvard Medical School, Boston, MA (L.B.G., M.E.K.); Department of Mathematics and Statistics, Boston University, Harvard Clinical Research Institute, Boston, MA (J.M., R.B.D.); Center for Gerontology and Health Care Research, Brown University, Providence, RI (S.E.C., J |
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| ContentType | Journal Article |
| Contributor | Bishop, W Robert Cleveland, Robert H Huh, Susanna Y Gordon, Catherine M Smoot, Leslie Miller, David T Nazarian, Ara Snyder, Brian D Moses, Marsha Gerhard-Herman, Marie Riley, Susan Liang, Marilyn Ullrich, Nicole J Silvera, V Michelle |
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| Copyright | 2015 INIST-CNRS 2014 American Heart Association, Inc. |
| Copyright_xml | – notice: 2015 INIST-CNRS – notice: 2014 American Heart Association, Inc. |
| CorporateAuthor | Progeria Clinical Trials Collaborative |
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| Keywords | Antineoplastic agent Skin disease Senescence Prognosis lamins Estimation Ageing Cardiovascular disease Survival Syndrome Kaplan-Meier method Dwarfism Vascular disease Lonafarnib Progeria Atherosclerosis prenylation Inhibitor Circulatory system aging Cardiology Kaplan-Meier estimate atherosclerosis progeria lonafarnib |
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| Snippet | Hutchinson-Gilford progeria syndrome is an ultrarare segmental premature aging disease resulting in early death from heart attack or stroke. There is no... |
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| SubjectTerms | Adolescent Adult Alkyl and Aryl Transferases - antagonists & inhibitors Atherosclerosis (general aspects, experimental research) Atherosclerosis - etiology Atherosclerosis - genetics Atherosclerosis - prevention & control Biological and medical sciences Blood and lymphatic vessels Cardiology. Vascular system Cause of Death Child Child, Preschool Clinical Trials as Topic - statistics & numerical data Cohort Studies Dimethylallyltranstransferase - antagonists & inhibitors Diphosphonates - administration & dosage Diphosphonates - pharmacology Diphosphonates - therapeutic use Diseases of the peripheral vessels. Diseases of the vena cava. Miscellaneous Drug Therapy, Combination Female Genes, Dominant Genotype Humans Hydroxymethylglutaryl-CoA Reductase Inhibitors - administration & dosage Hydroxymethylglutaryl-CoA Reductase Inhibitors - pharmacology Hydroxymethylglutaryl-CoA Reductase Inhibitors - therapeutic use Imidazoles - administration & dosage Imidazoles - pharmacology Imidazoles - therapeutic use Kaplan-Meier Estimate Lamin Type A Male Medical sciences Multicenter Studies as Topic - statistics & numerical data Nuclear Proteins - deficiency Nuclear Proteins - genetics Nuclear Proteins - metabolism Piperidines - administration & dosage Piperidines - pharmacology Piperidines - therapeutic use Pravastatin - administration & dosage Pravastatin - pharmacology Pravastatin - therapeutic use Progeria - complications Progeria - drug therapy Progeria - mortality Proportional Hazards Models Protein Precursors - deficiency Protein Precursors - genetics Protein Precursors - metabolism Protein Prenylation - drug effects Pyridines - administration & dosage Pyridines - pharmacology Pyridines - therapeutic use Treatment Outcome Young Adult Zoledronic Acid |
| Title | Impact of Farnesylation Inhibitors on Survival in Hutchinson-Gilford Progeria Syndrome |
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