Health-related quality of life benefits of arsenic trioxide in patients with non-high-risk acute promyelocytic leukemia are sustained over time: long-term results of the GIMEMA APL0406 trial.
Acute promyelocytic leukemia
Arsenic trioxide
Clinical trial
Patient-reported outcomes
Quality of life
Survivorship
Journal
Annals of hematology
ISSN: 1432-0584
Titre abrégé: Ann Hematol
Pays: Germany
ID NLM: 9107334
Informations de publication
Date de publication:
23 Oct 2024
23 Oct 2024
Historique:
received:
03
09
2024
accepted:
04
10
2024
medline:
23
10
2024
pubmed:
23
10
2024
entrez:
22
10
2024
Statut:
aheadofprint
Résumé
Very limited evidence is available on the long-term health-related quality of life (HRQoL) of patients with acute promyelocytic leukemia (APL) treated with arsenic trioxide (ATO). We performed an extended follow-up of the APL0406 randomized controlled trial to investigate HRQoL of patients treated with either ATO or chemotherapy. A secondary objective was to describe the prevalence of clinically important problems and symptoms of these patients by type of treatment. Overall, 117 patients were included in this analysis after a median follow-up of 10 years (IQR 8-11) since diagnosis. Of these, 60 (51.3%) were treated with ATO, and 57 (48.7%) with chemotherapy. A statistically significant and clinically relevant difference, favoring patients treated with ATO, was found in 2 of the 3 main prespecified EORTC QLQ-C30 scales, that is, cognitive functioning (∆ = 7.7; 95% CI 0.5 to 14.9; p = 0.036) and fatigue (∆ = -9.4; 95% CI -17.9 to -0.8; p = 0.031). The prevalence of clinically important problems and symptoms tended to be slightly higher in patients treated with chemotherapy. These findings suggest that previously observed HRQoL advantages of ATO therapy of patients included in the APL0406 trial are sustained over the long-term period. This study was registered at ClinicalTrials.gov (NCT03096496).
Identifiants
pubmed: 39438322
doi: 10.1007/s00277-024-06038-7
pii: 10.1007/s00277-024-06038-7
doi:
Banques de données
ClinicalTrials.gov
['NCT03096496']
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2024. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.
Références
Yilmaz M, Kantarjian H, Ravandi F (2021) Acute promyelocytic leukemia current treatment algorithms. Blood Cancer J 11(6):123
doi: 10.1038/s41408-021-00514-3
pubmed: 34193815
pmcid: 8245494
Burnett AK, Russell NH, Hills RK, Bowen D, Kell J, Knapper S et al (2015) Arsenic trioxide and all-trans retinoic acid treatment for acute promyelocytic leukaemia in all risk groups (AML17): results of a randomised, controlled, phase 3 trial. Lancet Oncol 16(13):1295–1305
doi: 10.1016/S1470-2045(15)00193-X
pubmed: 26384238
Lo-Coco F, Avvisati G, Vignetti M, Thiede C, Orlando SM, Iacobelli S et al (2013) Retinoic acid and arsenic trioxide for acute promyelocytic leukemia. N Engl J Med 369(2):111–121
doi: 10.1056/NEJMoa1300874
pubmed: 23841729
Platzbecker U, Avvisati G, Cicconi L, Thiede C, Paoloni F, Vignetti M et al (2017) Improved outcomes with retinoic acid and Arsenic Trioxide compared with retinoic acid and chemotherapy in Non-high-risk Acute promyelocytic leukemia: final results of the Randomized Italian-German APL0406 Trial. J Clin Oncol 35(6):605–612
doi: 10.1200/JCO.2016.67.1982
pubmed: 27400939
Guarnera L, Lehmann S, Döhner K, Döhner H, Platzbecker U, Russell NH et al (2023) Long-term outcome of 1296 patients with newly diagnosed with APL: a Harmony Alliance Study. Blood 142(Supplement 1):727
doi: 10.1182/blood-2023-185121
Miller KD, Nogueira L, Devasia T, Mariotto AB, Yabroff KR, Jemal A et al (2022) Cancer treatment and survivorship statistics, 2022. CA Cancer J Clin 72(5):409–436
doi: 10.3322/caac.21731
pubmed: 35736631
Ramsey I, de Rooij BH, Mols F, Corsini N, Horevoorts NJE, Eckert M et al (2019) Cancer survivors who fully participate in the PROFILES registry have better health-related quality of life than those who drop out. J Cancer Surviv 13(6):829–839
doi: 10.1007/s11764-019-00793-7
pubmed: 31493162
pmcid: 6881419
Ekels A, van de Poll-Franse LV, Posthuma EFM, Kieffer J, Issa DE, Koster A et al (2022) Persistent symptoms of fatigue, neuropathy and role-functioning impairment among indolent non-hodgkin lymphoma survivors: a longitudinal PROFILES registry study. Br J Haematol 197(5):590–601
doi: 10.1111/bjh.18139
pubmed: 35365860
Harrop JP, Dean JA, Paskett ED (2011) Cancer survivorship research: a review of the literature and summary of current NCI-designated cancer center projects. Cancer Epidemiol Biomarkers Prev 20(10):2042–2047
doi: 10.1158/1055-9965.EPI-11-0673
pubmed: 21980012
pmcid: 3191883
de Rooij BH, Ezendam NPM, Mols F, Vissers PAJ, Thong MSY, Vlooswijk CCP et al (2018) Cancer survivors not participating in observational patient-reported outcome studies have a lower survival compared to participants: the population-based PROFILES registry. Qual Life Res 27(12):3313–3324
doi: 10.1007/s11136-018-1979-0
pubmed: 30167937
pmcid: 6244797
Efficace F, Mandelli F, Avvisati G, Cottone F, Ferrara F, Bona ED et al (2014) Randomized Phase III Trial of Retinoic Acid and Arsenic Trioxide Versus Retinoic Acid and Chemotherapy in patients with Acute promyelocytic leukemia: Health-Related Quality-of-life outcomes. J Clin Oncol 32(30):3406–3412
doi: 10.1200/JCO.2014.55.3453
pubmed: 25245446
Efficace F, Platzbecker U, Breccia M, Cottone F, Carluccio P, Salutari P et al (2021) Long-term quality of life of patients with acute promyelocytic leukemia treated with arsenic trioxide vs chemotherapy. Blood Adv 5(21):4370–4379
doi: 10.1182/bloodadvances.2021004649
pubmed: 34529768
pmcid: 8579253
Efficace F, Cannella L, Breccia M, Olivieri J, Platzbecker U, Vignetti M (2021) Health-related quality of life in patients with acute promyelocytic leukemia: a systematic literature review. Expert Rev Hematol 14(7):645–654
doi: 10.1080/17474086.2021.1943352
pubmed: 34125642
Aaronson NK, Ahmedzai S, Bergman B et al (1993) The European Organization for Research and Treatment of Cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncology. J Natl Cancer Inst 85:365–376
doi: 10.1093/jnci/85.5.365
pubmed: 8433390
Fayers P, Aaronson N, Bjordal K et al (2001) EORTC QLQ-C30 Scoring Manual. 3rd Edition, Brussels, Belgium, EORTC Publications
Topp MS, Zimmerman Z, Cannell P, Dombret H, Maertens J, Stein A et al (2018) Health-related quality of life in adults with relapsed/refractory acute lymphoblastic leukemia treated with blinatumomab. Blood 131(26):2906–2914
doi: 10.1182/blood-2017-09-804658
pubmed: 29739753
pmcid: 6024638
Efficace F, Huls GA, Kicinski M, Van Der Velden WJ, Noppeney R, Chantepie S et al (2022) 10-Day decitabine Versus Intensive Chemotherapy Followed by Transplantation in Fit AML patients aged ≥ 60 years: Health-Related Quality of Life outcomes of the Randomized Phase III Trial AML21 of the EORTC Leukemia Group, Gimema, Celg, and Gmds-SG. Blood 140(Supplement 1):1281–1283
doi: 10.1182/blood-2022-166544
Ware JE Jr., Sherbourne CD (1992) The MOS 36-item short-form health survey (SF-36). I. conceptual framework and item selection. Med Care 30(6):473–483
doi: 10.1097/00005650-199206000-00002
pubmed: 1593914
Ware JE Jr., Kosinski M, Bayliss MS, McHorney CA, Rogers WH, Raczek A (1995) Comparison of methods for the scoring and statistical analysis of SF-36 health profile and summary measures: summary of results from the Medical outcomes Study. Med Care 33(4 Suppl):AS264–AS279
pubmed: 7723455
Smith SK, Mayer DK, Zimmerman S, Williams CS, Benecha H, Ganz PA et al (2013) Quality of life among long-term survivors of non-hodgkin lymphoma: a follow-up study. J Clin Oncol 31(2):272–279
doi: 10.1200/JCO.2011.40.6249
pubmed: 23213093
Efficace F, Baccarani M, Breccia M, Alimena G, Rosti G, Cottone F et al (2011) Health-related quality of life in chronic myeloid leukemia patients receiving long-term therapy with imatinib compared with the general population. Blood 118(17):4554–4560
doi: 10.1182/blood-2011-04-347575
pubmed: 21750313
Yellen SB, Cella DF, Webster K, Blendowski C, Kaplan E (1997) Measuring fatigue and other anemia-related symptoms with the Functional Assessment of Cancer Therapy (FACT) measurement system. J Pain Symptom Manage 13(2):63–74
doi: 10.1016/S0885-3924(96)00274-6
pubmed: 9095563
Cella D (1997) The Functional Assessment of Cancer Therapy-Anemia (FACT-An) Scale: a new tool for the assessment of outcomes in cancer anemia and fatigue. Semin Hematol 34(3 Suppl 2):13–19
pubmed: 9253779
Cocks K, King MT, Velikova G, Martyn St-James M, Fayers PM, Brown JM (2011) Evidence-based guidelines for determination of sample size and interpretation of the European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire Core 30. J Clin Oncol 29(1):89–96
doi: 10.1200/JCO.2010.28.0107
pubmed: 21098316
Sloan J, Symonds T, Vargas-Chanes D, Fridley B (2003) Practical guidelines for assessing the clinical significance of health-related quality of life changes within clinical trials. Drug Inform J 37(1):23–31
doi: 10.1177/009286150303700105
Colangelo KJ, Pope JE, Peschken C (2009) The minimally important difference for patient reported outcomes in systemic lupus erythematosus including the HAQ-DI, pain, fatigue, and SF-36. J Rheumatol 36(10):2231–2237
doi: 10.3899/jrheum.090193
pubmed: 19723907
Sekhon S, Pope J, Baron M (2010) The minimally important difference in clinical practice for patient-centered outcomes including health assessment questionnaire, fatigue, pain, sleep, global visual analog scale, and SF-36 in scleroderma. J Rheumatol 37(3):591–598
doi: 10.3899/jrheum.090375
pubmed: 20080913
Cella D, Eton DT, Lai JS, Peterman AH, Merkel DE (2002) Combining anchor and distribution-based methods to derive minimal clinically important differences on the Functional Assessment of Cancer Therapy (FACT) anemia and fatigue scales. J Pain Symptom Manage 24(6):547–561
doi: 10.1016/S0885-3924(02)00529-8
pubmed: 12551804
Giesinger JM, Loth FLC, Aaronson NK, Arraras JI, Caocci G, Efficace F et al (2020) Thresholds for clinical importance were established to improve interpretation of the EORTC QLQ-C30 in clinical practice and research. J Clin Epidemiol 118:1–8
doi: 10.1016/j.jclinepi.2019.10.003
pubmed: 31639445
Yin XJ, Wang R, Shen HS, Jin J, Zhu HH (2023) At what point are long-term (> 5 years) survivors of APL Safe? A study from the SEER database. Cancers (Basel). 15(3)
Zhu H, Hu J, Chen L, Zhou W, Li X, Wang L et al (2016) The 12-year follow-up of survival, chronic adverse effects, and retention of arsenic in patients with acute promyelocytic leukemia. Blood 128(11):1525–1528
doi: 10.1182/blood-2016-02-699439
pubmed: 27402972
Sommer K, Vignetti M, Cottone F, Breccia M, Annibali O, Luppi M et al (2022) Acute promyelocytic leukaemia long-term survivors: higher fatigue and greater overall symptom burden. BMJ Support Palliat Care 12(2):182–186
doi: 10.1136/bmjspcare-2020-002281
pubmed: 32680895
Oerlemans S, Mols F, Issa DE, Pruijt JH, Peters WG, Lybeert M et al (2013) A high level of fatigue among long-term survivors of Non-hodgkin’s lymphoma: results from the longitudinal population-based PROFILES registry in the south of the Netherlands. Haematologica 98(3):479–486
doi: 10.3324/haematol.2012.064907
pubmed: 22929981
pmcid: 3659932
Kiserud CE, Lockmer S, Baerug I, Dahl AA, Kimby E, Østenstad B (2023) Health-related quality of life and chronic fatigue in long-term survivors of indolent lymphoma - a comparison with normative data. Leuk Lymphoma 64(2):349–355
doi: 10.1080/10428194.2022.2142050
pubmed: 36342349
Sparano F, Voso MT, Venditti A, Giesinger JM, Baldi T, Breccia M et al (2024) Financial toxicity and health-related quality of life in long-term survivors of acute promyelocytic leukaemia. BMJ Support Palliat Care. https://doi.org/10.1136/spcare-2024-004924