{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,2,21]],"date-time":"2025-02-21T03:01:44Z","timestamp":1740106904724,"version":"3.37.3"},"reference-count":22,"publisher":"Springer Science and Business Media LLC","issue":"6","license":[{"start":{"date-parts":[[2024,4,5]],"date-time":"2024-04-05T00:00:00Z","timestamp":1712275200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2024,4,5]],"date-time":"2024-04-05T00:00:00Z","timestamp":1712275200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Ann Hematol"],"published-print":{"date-parts":[[2024,6]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Acquired aplastic anemia (AA) is a rare form of immune-mediated bone marrow failure, which can result in life-threatening infections or bleeding if left untreated. Treatment consists of either immune suppressive therapy (IST) or allogeneic stem cell transplantation (alloHSCT). While considerable research has been published regarding survival, response rate and toxicity of both treatments, knowledge on the impact on quality of life (QoL) is scarce. We used the recently developed AA-specific QoL questionnaire (QLQ-AA\/PNH-54) to evaluate QoL in a single center cohort of AA patients who were successfully treated with IST. The 54 questions represent 12 different QoL domains. Results were analyzed for all patients and grouped based on hematologic response (complete response (CR) or partial response (PR)). Thirty-six successfully treated adult patients (15 in CR, 21 in PR) completed the questionnaire (median age 54\u00a0years, range 21\u201371; median time since last IST 5\u00a0years, range 0\u201341). Fatigue was experienced by 83% of patients. Even though total QoL scores did not significantly differ between patients with PR and CR (105 vs 92, p-value 0,17) there appeared to be a trend towards higher scores in patients with PR, especially in domains concerning psychological wellbeing. This trend was most clear in the domains fear of progression (2,12 in PR patients vs 1,73 in CR patients; p-value 0,08) and role functioning (2,22 vs 1,88; p-value 0,07). In conclusion, patients with AA continue to experience psychological and physical effects despite successful IST.<\/jats:p>","DOI":"10.1007\/s00277-024-05731-x","type":"journal-article","created":{"date-parts":[[2024,4,5]],"date-time":"2024-04-05T14:01:34Z","timestamp":1712325694000},"page":"2113-2121","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Quality of life after immune suppressive therapy in aplastic anemia"],"prefix":"10.1007","volume":"103","author":[{"ORCID":"https:\/\/orcid.org\/0009-0003-9577-6791","authenticated-orcid":false,"given":"Iris N.","family":"Lommerse","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Chris","family":"Hinnen","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Liesbeth M.","family":"van Vliet","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Beke","family":"Schubert","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jens","family":"Panse","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Constantijn J. M.","family":"Halkes","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jennifer M.-L.","family":"Tjon","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2024,4,5]]},"reference":[{"issue":"17","key":"5731_CR1","doi-asserted-by":"publisher","first-page":"1643","DOI":"10.1056\/NEJMra1413485","volume":"379","author":"N Young","year":"2018","unstructured":"Young N (2018) Aplastic anemia. N Engl J Med 379(17):1643\u20131656. https:\/\/doi.org\/10.1056\/NEJMra1413485","journal-title":"N Engl J Med"},{"issue":"7","key":"5731_CR2","doi-asserted-by":"publisher","DOI":"10.1136\/bmjopen-2014-005039","volume":"4","author":"F Peinemann","year":"2014","unstructured":"Peinemann F, Labeit A (2014) Stem cell transplantation of matched sibling donors compared with immunosuppressive therapy for acquired severe aplastic anaemia: a Cochrane systematic review. BMJ Open 4(7):e005039. https:\/\/doi.org\/10.1136\/bmjopen-2014-005039","journal-title":"BMJ Open"},{"issue":"15","key":"5731_CR3","doi-asserted-by":"publisher","first-page":"2020","DOI":"10.1182\/bloodadvances.2018021162","volume":"2","author":"GE Georges","year":"2018","unstructured":"Georges GE et al (2018) Severe aplastic anemia: allogeneic bone marrow transplantation as first-line treatment. Blood Adv 2(15):2020\u20132028. https:\/\/doi.org\/10.1182\/bloodadvances.2018021162","journal-title":"Blood Adv"},{"issue":"2","key":"5731_CR4","doi-asserted-by":"publisher","first-page":"371","DOI":"10.1111\/j.1365-2141.1993.tb03181.x","volume":"85","author":"H Schrezenmeier","year":"1993","unstructured":"Schrezenmeier H et al (1993) Relapse of aplastic anaemia after immunosuppressive treatment: a report from the European Bone Marrow Transplantation Group SAA Working Party. Br J Haematol 85(2):371\u2013377. https:\/\/doi.org\/10.1111\/j.1365-2141.1993.tb03181.x","journal-title":"Br J Haematol"},{"issue":"10","key":"5731_CR5","doi-asserted-by":"publisher","first-page":"e382","DOI":"10.3324\/haematol.2017.171215","volume":"102","author":"J Van Der Hem","year":"2017","unstructured":"Van Der Hem J et al (2017) Long-term risk of cancer development in adult patients with idiopathic aplastic anemia after treatment with anti-thymocyte globulin. Haematologica 102(10):e382\u2013e383. https:\/\/doi.org\/10.3324\/haematol.2017.171215","journal-title":"Haematologica"},{"issue":"2","key":"5731_CR6","doi-asserted-by":"publisher","first-page":"187","DOI":"10.1111\/bjh.13853","volume":"172","author":"SB Killick","year":"2015","unstructured":"Killick SB et al (2015) Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol 172(2):187\u2013207. https:\/\/doi.org\/10.1111\/bjh.13853","journal-title":"Br J Haematol"},{"issue":"6","key":"5731_CR7","doi-asserted-by":"publisher","first-page":"954","DOI":"10.1111\/bjh.17403","volume":"194","author":"P Scheinberg","year":"2021","unstructured":"Scheinberg P (2021) Acquired severe aplastic anaemia: How medical therapy evolved in the 20th and 21st centuries. Br J Haematol 194(6):954\u2013969. https:\/\/doi.org\/10.1111\/bjh.17403","journal-title":"Br J Haematol"},{"key":"5731_CR8","unstructured":"Tjon, JM-L et al. (2019) Diagnostiek en behandeling verworven aplastische anemie bij volwassenen. [guideline] Retrieved from https:\/\/hematologienederland.nl\/wp-content\/uploads\/2019\/07\/richtlijn_verworven_aplastische_anemie_2019_definitief.pdf. Accessed\u00a012 June 2023"},{"issue":"9","key":"5731_CR9","doi-asserted-by":"publisher","first-page":"714","DOI":"10.1093\/oncolo\/oyac122","volume":"27","author":"CD Bergerot","year":"2022","unstructured":"Bergerot CD et al (2022) Patient-reported outcomes in early phase clinical trials: An opportunity to actively promote patient-centered care. Oncologist 27(9):714\u2013715. https:\/\/doi.org\/10.1093\/oncolo\/oyac122","journal-title":"Oncologist"},{"key":"5731_CR10","doi-asserted-by":"publisher","unstructured":"Jim, H, Oswald, L (2022) Patient-reported outcomes: Important data in early trials of novel therapies. The Lancet Haematology, 9(12). https:\/\/doi.org\/10.1016\/s2352-3026(22)00295-2","DOI":"10.1016\/s2352-3026(22)00295-2"},{"issue":"1","key":"5731_CR11","doi-asserted-by":"publisher","first-page":"47","DOI":"10.1007\/s00277-004-0930-3","volume":"84","author":"R Viollier","year":"2004","unstructured":"Viollier R et al (2004) Quality-adjusted survival analysis shows differences in outcome after immunosuppression or bone marrow transplantation in aplastic anemia. Ann Hematol 84(1):47\u201355. https:\/\/doi.org\/10.1007\/s00277-004-0930-3","journal-title":"Ann Hematol"},{"issue":"9","key":"5731_CR12","doi-asserted-by":"publisher","first-page":"963","DOI":"10.1016\/j.scib.2022.01.024","volume":"67","author":"L Xu","year":"2022","unstructured":"Xu L et al (2022) Long-term follow-up of haploidentical transplantation in relapsed\/refractory severe aplastic anemia: a multicenter prospective study. Science Bulletin 67(9):963\u2013970. https:\/\/doi.org\/10.1016\/j.scib.2022.01.024","journal-title":"Science Bulletin"},{"issue":"2","key":"5731_CR13","doi-asserted-by":"publisher","first-page":"543","DOI":"10.1002\/cam4.1953","volume":"8","author":"CP Escalante","year":"2019","unstructured":"Escalante CP et al (2019) Fatigue, symptom burden, and health-related quality of life in patients with myelodysplastic syndrome, aplastic anemia, and paroxysmal nocturnal hemoglobinuria. Cancer Med 8(2):543\u2013553. https:\/\/doi.org\/10.1002\/cam4.1953","journal-title":"Cancer Med"},{"issue":"2","key":"5731_CR14","doi-asserted-by":"publisher","first-page":"171","DOI":"10.1007\/s00277-016-2867-8","volume":"96","author":"M Groth","year":"2017","unstructured":"Groth M et al (2017) Development of a disease-specific quality of life questionnaire for patients with aplastic anemia and\/or paroxysmal nocturnal hemoglobinuria (QLQ-AA\/PNH)\u2014report on phases I and II. Ann Hematol 96(2):171\u2013181. https:\/\/doi.org\/10.1007\/s00277-016-2867-8","journal-title":"Ann Hematol"},{"issue":"7","key":"5731_CR15","doi-asserted-by":"publisher","first-page":"1547","DOI":"10.1007\/s00277-019-03681(2019)","volume":"98","author":"C Niedeggen","year":"2019","unstructured":"Niedeggen C et al (2019) Design and development of a disease-specific quality of life tool for patients with aplastic anaemia and\/or paroxysmal nocturnal haemoglobinuria (QLQ-AA\/PNH)\u2014a report on phase III. Ann Hematol 98(7):1547\u20131559. https:\/\/doi.org\/10.1007\/s00277-019-03681(2019","journal-title":"Ann Hematol"},{"key":"5731_CR16","unstructured":"EORTC quality of life group guidelines for module development (2021) [guideline] Retrieved from https:\/\/www.eortc.org\/app\/uploads\/sites\/2\/2022\/07\/Module-Guidelines-Version-5-FINAL.pdf. Accessed\u00a07 Oct 2023"},{"key":"5731_CR17","doi-asserted-by":"publisher","first-page":"327","DOI":"10.2147\/jbm.s339660","volume":"13","author":"B Fattizzo","year":"2022","unstructured":"Fattizzo B, Cavallaro F, Oliva EN, Barcellini W (2022) Managing fatigue in patients with paroxysmal nocturnal hemoglobinuria: A patient-focused perspective. J Blood Med 13:327\u2013335. https:\/\/doi.org\/10.2147\/jbm.s339660","journal-title":"J Blood Med"},{"key":"5731_CR18","doi-asserted-by":"publisher","DOI":"10.1016\/j.lrr.2020.100224","volume":"14","author":"K Sakai","year":"2020","unstructured":"Sakai K (2020) Even mild hemolysis in paroxysmal nocturnal hemoglobinuria could severely compromise the quality of life due to long-term sustained intolerant fatigue. Leukemia Res Rep 14:100224. https:\/\/doi.org\/10.1016\/j.lrr.2020.100224","journal-title":"Leukemia Res Rep"},{"issue":"1","key":"5731_CR19","doi-asserted-by":"publisher","first-page":"e75","DOI":"10.1097\/SLA.0000000000004078","volume":"275","author":"VM Meyer","year":"2022","unstructured":"Meyer VM et al (2022) Global Overview of Response Rates in Patient and Health Care Professional Surveys in Surgery: A Systematic Review. Annals of Surgery, [online] 275(1):e75. https:\/\/doi.org\/10.1097\/SLA.0000000000004078","journal-title":"Annals of Surgery, [online]"},{"issue":"5","key":"5731_CR20","doi-asserted-by":"publisher","first-page":"677","DOI":"10.1111\/j.1365-2354.2012.01367.x","volume":"21","author":"A Hinz","year":"2012","unstructured":"Hinz A et al (2012) Is it useful to calculate sum scores of the quality of life questionnaire EORTC QLQ-C30? Eur J Cancer Care 21(5):677\u2013683. https:\/\/doi.org\/10.1111\/j.1365-2354.2012.01367.x","journal-title":"Eur J Cancer Care"},{"key":"5731_CR21","doi-asserted-by":"publisher","first-page":"2559","DOI":"10.1007\/s11136-015-0991-x","volume":"24","author":"RM Ciconelli","year":"2015","unstructured":"Ciconelli RM et al (2015) Brazilian urban population norms derived from the health-related quality of life SF-6D. Qual Life Res 24:2559\u20132564. https:\/\/doi.org\/10.1007\/s11136-015-0991-x","journal-title":"Qual Life Res"},{"issue":"1","key":"5731_CR22","doi-asserted-by":"publisher","first-page":"42","DOI":"10.1080\/15350770.2018.1500330","volume":"17","author":"S Villas-Boas","year":"2019","unstructured":"Villas-Boas S et al (2019) Predictors of Quality of Life in Different Age Groups Across Adulthood. J Intergenerational Relationships 17(1):42\u201357. https:\/\/doi.org\/10.1080\/15350770.2018.1500330","journal-title":"J Intergenerational Relationships"}],"container-title":["Annals of Hematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00277-024-05731-x.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s00277-024-05731-x\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00277-024-05731-x.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,5,13]],"date-time":"2024-05-13T06:06:07Z","timestamp":1715580367000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s00277-024-05731-x"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,4,5]]},"references-count":22,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2024,6]]}},"alternative-id":["5731"],"URL":"https:\/\/doi.org\/10.1007\/s00277-024-05731-x","relation":{},"ISSN":["0939-5555","1432-0584"],"issn-type":[{"type":"print","value":"0939-5555"},{"type":"electronic","value":"1432-0584"}],"subject":[],"published":{"date-parts":[[2024,4,5]]},"assertion":[{"value":"31 October 2023","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"26 March 2024","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"5 April 2024","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"The authors have no competing interests to disclose.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}},{"value":"This study was performed in line with the principles of the Declaration of Helsinki. Approval was granted by the LUMC Biobank Review Committee (RP23.045).","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}},{"value":"Informed consent for collecting data was obtained from all patients participating in this study. All medical data used for analysis was gathered for regular evaluation of care.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent to participate"}}]}}