Cysteamine (Cystagon®) adherence in patients with cystinosis in Spain: successful in children and a challenge in adolescents and adults
| Autor: | Maria Luisa Justa, José F. Crespo, Adela Urisarri, Francisco Llamas, Rafael Bedoya, Julia Vara, Gema Ariceta, Juan Antonio Camacho, Enrique Lara, Luis Antonio Jiménez del Cerro, Blanca Valenciano, J. Bravo, Miguel A. Muñoz, Fernando Santos, Rafael Romero, Alberto Bueno, M. Calvo, Víctor García-Nieto, Antonio Daza, Federico Oppenheimer, Carmen Cantarell, Maria José Sanahuja |
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| Přispěvatelé: | [Ariceta,G, Lara,E, Cantarell,C] Hospital Universitari Vall d’ Hebron, Barcelona, Spain. [Ariceta,G] Hospital Universitario Cruces, Bilbao, Spain. [Camacho,JA] Hospital Sant Joan de Déu, Barcelona, Spain. [Oppenheimer ,F] Hospital Universitari Clínic, Barcelona, Spain. [Vara,J] Hospital Universitario 12 de Octubre, Madrid, Spain. [Santos ,F] Hospital Universitario Central de Asturias . Universidad de Oviedo, Oviedo, Spain. [Muñoz ,MA] Hospital Universitario Virgen de la Salud, Toledo, Spain. [Gil Calvo,M, Romero,R] Complexo Hospitalario Universitario de Santiago de Compostela, Santiago de Compostela, Spain. [Valenciano,B] Hospital Universitario Materno-Infantil Las Palmas, Las Palmas de Gran Canaria, Spain. [García-Nieto,V] Hospital Universitario Ntra. Sra. de la Candelaria, Santa Cruz de Tenerife, Spain. [Sanahuja,MJ] Hospital Universitario La Fe, Valencia, Spain. [Crespo,J] Hospital Universitario Dr Peset, Valencia, Spain. [Justa,ML] Hospital Universitario Miguel Servet, Zaragoza, Spain. [Urisarri,A] Hospital Arquitecto Marcide, Ferrol, Spain. [Bedoya,R] Hospital Universitario Virgen del Rocío, Sevilla, Spain. [Bueno,A] Hospital Universitario Materno-Infantil, Materno-Infantil Carlos Haya, Málaga, Spain. [Daza,A] Complejo Hospitalario de Torrecárdenas, Almería, Spain. [Bravo,J] Hospital Universitario Virgen de Las Nieves, Granada, Spain. [Llamas,F] Hospital General de Albacete, Albacete, Spain. [Jiménez Del Cerro,LA] Hospital General Universitario de Alicante, Alicante, Spain., This study has been supported by A.C. Nielsen Company S.L.and Orphan Europe S.L.U |
| Rok vydání: | 2015 |
| Předmět: |
Male
Nephrology Health Knowledge Attitudes Practice Pediatrics Anatomy::Urogenital System::Urinary Tract::Kidney [Medical Subject Headings] Autocuidado Cystinosis España Analytical Diagnostic and Therapeutic Techniques and Equipment::Therapeutics::Renal Replacement Therapy::Kidney Transplantation [Medical Subject Headings] Disease Cohort Studies Organisms::Eukaryota::Animals::Chordata::Vertebrates::Mammals::Primates::Haplorhini::Catarrhini::Hominidae::Humans [Medical Subject Headings] chemistry.chemical_compound Named Groups::Persons::Parents::Mothers [Medical Subject Headings] Interquartile range Chronic Kidney Disease Trasplante de riñón Named Groups::Persons::Occupational Groups::Health Personnel::Physicians [Medical Subject Headings] adherence Young adult Child Psychiatry and Psychology::Behavior and Behavior Mechanisms::Behavior::Health Behavior::Patient Compliance [Medical Subject Headings] Geographicals::Geographic Locations::Europe::Spain [Medical Subject Headings] Phenomena and Processes::Musculoskeletal and Neural Physiological Phenomena::Nervous System Physiological Phenomena::Nervous System Physiological Processes::Sensation::Smell [Medical Subject Headings] Pronóstico transition Prognosis Named Groups::Persons::Parents::Fathers [Medical Subject Headings] adherence cysteamine cystinosis prognosis transition Child Preschool Named Groups::Persons::Age Groups::Adolescent [Medical Subject Headings] Transition Absenteeism Chemicals and Drugs::Organic Chemicals::Amines::Ethylamines::Mercaptoethylamines::Cysteamine [Medical Subject Headings] Female Estudios de seguimiento Cohort study Adult medicine.medical_specialty Adolescent Cysteamine Check Tags::Male [Medical Subject Headings] Cistinosis CLINICAL SCIENCE Medication Adherence Young Adult Diseases::Congenital Hereditary and Neonatal Diseases and Abnormalities::Genetic Diseases Inborn::Metabolism Inborn Errors::Lysosomal Storage Diseases::Cystinosis [Medical Subject Headings] Internal medicine Analytical Diagnostic and Therapeutic Techniques and Equipment::Investigative Techniques::Epidemiologic Methods::Epidemiologic Study Characteristics as Topic::Epidemiologic Studies::Cohort Studies::Longitudinal Studies::Follow-Up Studies [Medical Subject Headings] Named Groups::Persons::Age Groups::Adult [Medical Subject Headings] medicine Humans Cooperación del paciente Named Groups::Persons::Parents [Medical Subject Headings] Cystine Depleting Agents Named Groups::Persons::Age Groups::Child [Medical Subject Headings] Analytical Diagnostic and Therapeutic Techniques and Equipment::Therapeutics::Self Care [Medical Subject Headings] Psychiatry and Psychology::Psychological Phenomena and Processes::Psychology Applied::Psychology Industrial::Absenteeism [Medical Subject Headings] Transplantation business.industry Infant Newborn Infant Psychiatry and Psychology::Behavior and Behavior Mechanisms::Motivation [Medical Subject Headings] medicine.disease Self Care chemistry Spain Adherence Psychiatry and Psychology::Behavior and Behavior Mechanisms::Emotions [Medical Subject Headings] Absentismo Cisteamina Analytical Diagnostic and Therapeutic Techniques and Equipment::Diagnosis::Prognosis [Medical Subject Headings] Olfato business |
| Zdroj: | NEPHROLOGY DIALYSIS TRANSPLANTATION r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu instname r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe r-FSJD: Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu Fundació Sant Joan de Déu Nephrology Dialysis Transplantation |
| ISSN: | 0931-0509 |
| Popis: | Cysteamine has improved survival and prognosis in cystinosis. Increasing numbers of patients reach adulthood and face new challenges such as compliance that wanes over time. The aim of this study was to evaluate adherence to cysteamine treatment in a group of cystinotic patients in Spain in an attempt to identify potential therapy pitfalls and improve the overall care of affected individuals. Despite the impact of cysteamine on prognosis, there is a paucity of data regarding adherence. Thirty-four cystinotic patients (21 male) 38% a parts per thousand yen18 years were enrolled in a voluntary, anonymous survey. Replies were obtained from patients (15/34), mothers (11/34), fathers (4/34) and both parents (4/34). Patient age (median and interquartile range) at diagnosis was 1 year (0.57-1), and patient age at CystagonA (R) initiation was also 1 year (0.8-1.8). Sixteen (47%) were kidney transplant (KTx) recipients; six were retransplanted. Age at first KTx 10 years (8.7-13.7). Patient understanding of multiorgan involvement in cystinosis: 4.1 organs reported; eye 97% and kidney 91%. Cysteamine was given by mother (100%) and father (83%) in < 11 year olds, or self-administered (94%) in a parts per thousand yen11 year olds. Four daily doses in 89% versus 56% in < 11 year olds or a parts per thousand yen11 year olds, with fixed schedule in 94% versus 50% in < 11 or a parts per thousand yen11 year olds and progressive loss of reminders over time. Furthermore, 44% complained of unpleasant smell. Motivation for treatment compliance was 100% versus 40% in < 11 versus a parts per thousand yen11 year olds, respectively. Disease impact in patients < 18 years is as follows: school (29%), social (14%), 'feeling different' (10%); in patients a parts per thousand yen18 years: 'feeling different' (62%), professional (39%) and job absenteeism (31%). Referring physician: paediatric nephrologist (94%) and nephrologist (63%) in < 11 versus a parts per thousand yen11 year olds. Ophthalmological follow-up: 83% versus 38% in < 11 versus a parts per thousand yen11 year olds. Patient opinion of physician expertise: paediatric nephrologist (94%) and nephrologist (44%). New treatment options (65%) and better information (42%) were demanded to improve adherence. Treatment with Cystagon is effective in young patients. However, adherence diminishes over time in adolescents and adults despite disease impact. Strategies such as better information on the disease, patient self-care promotion and facilitated transition to adult healthcare services are required to improve compliance and the clinical management of cystinosis. |
| Databáze: | OpenAIRE |
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