{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,1]],"date-time":"2026-08-01T12:54:17Z","timestamp":1785588857994,"version":"3.56.0"},"reference-count":36,"publisher":"FapUNIFESP (SciELO)","issue":"9","license":[{"start":{"date-parts":[[2019,1,1]],"date-time":"2019-01-01T00:00:00Z","timestamp":1546300800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2019,1,1]],"date-time":"2019-01-01T00:00:00Z","timestamp":1546300800000},"content-version":"am","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2019,1,1]],"date-time":"2019-01-01T00:00:00Z","timestamp":1546300800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Cad. Sa\u00fade P\u00fablica"],"abstract":"<jats:p>Resumo: Estimativas apontam que h\u00e1 mais de 7 mil doen\u00e7as raras j\u00e1 identificadas, que representam de 6 a 10% de todas as doen\u00e7as no mundo. No Brasil, considera-se doen\u00e7a rara aquela que afeta at\u00e9 65 pessoas em cada 100 mil indiv\u00edduos. Quantificar os custos para as fam\u00edlias de pacientes com essas condi\u00e7\u00f5es e o seu comprometimento sobre a renda fornece informa\u00e7\u00f5es capazes de apoiar as pol\u00edticas p\u00fablicas destinadas a esses pacientes. O objetivo deste estudo foi estimar o custo e a perda de renda sob a perspectiva das fam\u00edlias de crian\u00e7as e adolescentes com fibrose c\u00edstica, mucopolissacaridoses e osteog\u00eanese imperfeita. Foi realizado com 99 fam\u00edlias de pacientes atendidos em um hospital de refer\u00eancia nacional em doen\u00e7as raras no Rio de Janeiro, mediante relato do cuidador principal. A an\u00e1lise descritiva dos dados mostrou que o custo mediano direto n\u00e3o m\u00e9dico para as fam\u00edlias foi de R$ 2.156,56 para fibrose c\u00edstica, R$ 1.060,00 para mucopolissacaridoses e R$ 1.908,00 para osteog\u00eanese imperfeita. A perda de renda superou 100% para as tr\u00eas condi\u00e7\u00f5es analisadas. Um total de 54% das fam\u00edlias n\u00e3o recebem benef\u00edcios assistenciais. A estimativa de coping costs indicou que 69% das fam\u00edlias acessaram empr\u00e9stimos e 22,5% venderam bens para lidar com os custos do curso do tratamento. Foram verificados gastos catastr\u00f3ficos para as fam\u00edlias das tr\u00eas doen\u00e7as analisadas. Os resultados trazem \u00e0 tona um tema que descortina custos pouco estimados, n\u00e3o somente no campo das doen\u00e7as raras. S\u00e3o resultados que indicam uma carga importante sobre a renda das fam\u00edlias. \u00c9 importante incorporar estudos de tal natureza na discuss\u00e3o do financiamento, da incorpora\u00e7\u00e3o de novas tecnologias e da oferta de servi\u00e7os de sa\u00fade.<\/jats:p>","DOI":"10.1590\/0102-311x00180218","type":"journal-article","created":{"date-parts":[[2019,9,7]],"date-time":"2019-09-07T19:21:34Z","timestamp":1567884094000},"source":"Crossref","is-referenced-by-count":18,"title":["Cuidado complexo, custo elevado e perda de renda: o que n\u00e3o \u00e9 raro para as fam\u00edlias de crian\u00e7as e adolescentes com condi\u00e7\u00f5es de sa\u00fade raras"],"prefix":"10.1590","volume":"35","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7568-5014","authenticated-orcid":false,"given":"M\u00e1rcia","family":"Pinto","sequence":"first","affiliation":[{"name":"Funda\u00e7\u00e3o Oswaldo Cruz,  Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9963-9290","authenticated-orcid":false,"given":"Adelino","family":"Madureira","sequence":"additional","affiliation":[{"name":"Funda\u00e7\u00e3o Oswaldo Cruz,  Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6610-9357","authenticated-orcid":false,"given":"Let\u00edcia Baptista de Paula","family":"Barros","sequence":"additional","affiliation":[{"name":"Funda\u00e7\u00e3o Oswaldo Cruz,  Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3363-4232","authenticated-orcid":false,"given":"Marcos","family":"Nascimento","sequence":"additional","affiliation":[{"name":"Funda\u00e7\u00e3o Oswaldo Cruz,  Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9456-3319","authenticated-orcid":false,"given":"Ana Carolina Carioca da","family":"Costa","sequence":"additional","affiliation":[{"name":"Funda\u00e7\u00e3o Oswaldo Cruz,  Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3653-7956","authenticated-orcid":false,"given":"Nicole Velloso de","family":"Oliveira","sequence":"additional","affiliation":[{"name":"Funda\u00e7\u00e3o Oswaldo Cruz,  Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2449-1142","authenticated-orcid":false,"given":"Lidianne","family":"Albernaz","sequence":"additional","affiliation":[{"name":"Funda\u00e7\u00e3o Oswaldo Cruz,  Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8937-7474","authenticated-orcid":false,"given":"Daniel de Souza","family":"Campos","sequence":"additional","affiliation":[{"name":"Funda\u00e7\u00e3o Oswaldo Cruz,  Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7726-4301","authenticated-orcid":false,"given":"Dafne Dain Gandelman","family":"Horovitz","sequence":"additional","affiliation":[{"name":"Funda\u00e7\u00e3o Oswaldo Cruz,  Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1617-8033","authenticated-orcid":false,"given":"Antilia Janu\u00e1ria","family":"Martins","sequence":"additional","affiliation":[{"name":"Funda\u00e7\u00e3o Oswaldo Cruz,  Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7199-3797","authenticated-orcid":false,"given":"Martha Cristina Nunes","family":"Moreira","sequence":"additional","affiliation":[{"name":"Funda\u00e7\u00e3o Oswaldo Cruz,  Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"530","published-online":{"date-parts":[[2019]]},"reference":[{"key":"ref1","series-title":"Rare disease database"},{"key":"ref2","series-title":"Rare diseases and related terms"},{"key":"ref3","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1111\/cts.12501","article-title":"Progress in rare diseases research 2010-2016: an IRDiRC perspective","volume":"11","author":"Dawkins HJS","year":"2018","journal-title":"Clin Transl Sci"},{"key":"ref4","article-title":"Portaria GM n\u00ba 199, de 30 de janeiro de 2014. 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