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.

Background: Acute promyelocytic leukaemia is a chemotherapy-sensitive subgroup of acute myeloid leukaemia characterised by the presence of the PML-RARA fusion transcript. The present standard of care, chemotherapy and all-trans retinoic acid (ATRA), results in a high proportion of patients being cur...

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Publicado en:Lancet Oncology Vol. 16; no. 13; pp. 1295 - 1306
Autores principales: Burnett, Alan K, Russell, Nigel H, Hills, Robert K, Bowen, David, Kell, Jonathan, Knapper, Steve, Morgan, Yvonne G, Lok, Jennie, Grech, Angela, Jones, Gail, Khwaja, Asim, Friis, Lone, McMullin, Mary Frances, Hunter, Ann, Clark, Richard E, Grimwade, David
Formato: research randomized controlled trial Journal Article
Publicado: Elsevier B.V. Oct2015
Acceso en línea:Ver este registro en EBSCOhost
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      dt: Oct2015
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      ed: 1077-4114 (Print)
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      pub: Elsevier B.V.
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        10.1016/S1470-2045(15)00193-X
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        atl: 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.
      aug:
        au:
          Burnett, Alan K
          Russell, Nigel H
          Hills, Robert K
          Bowen, David
          Kell, Jonathan
          Knapper, Steve
          Morgan, Yvonne G
          Lok, Jennie
          Grech, Angela
          Jones, Gail
          Khwaja, Asim
          Friis, Lone
          McMullin, Mary Frances
          Hunter, Ann
          Clark, Richard E
          Grimwade, David
        affil: Department of Haematology Cardiff University School of Medicine, Cardiff, UK
      sug:
        subj:
          Tretinoin Therapeutic Use
          Leukemia, Promyelocytic, Acute Drug Therapy
          Antineoplastic Agents, Combined Therapeutic Use
          Oxides Therapeutic Use
          Arsenicals Therapeutic Use
          Idarubicin Therapeutic Use
          Polymerase Chain Reaction
          Leukemia, Promyelocytic, Acute Diagnosis
          Leukemia, Promyelocytic, Acute
          Adult
          Human
          Leukemia, Promyelocytic, Acute Mortality
          Treatment Outcomes
          Proteins
          Denmark
          Middle Age
          Great Britain
          Quality of Life
          Female
          New Zealand
          Antineoplastic Agents, Combined Adverse Effects
          Tretinoin Adverse Effects
          Time Factors
          Arsenicals Adverse Effects
          Oxides Adverse Effects
          Young Adult
          Adolescence
          Leukocyte Count
          Kaplan-Meier Estimator
          Idarubicin Adverse Effects
          Male
          Validation Studies
          Comparative Studies
          Evaluation Research
          Multicenter Studies
          Randomized Controlled Trials
          Questionnaires
          Funding Source
          Adult: 19-44 years
          Middle Aged: 45-64 years
          Adolescent: 13-18 years
          Female
          Male
      ab: Background: Acute promyelocytic leukaemia is a chemotherapy-sensitive subgroup of acute myeloid leukaemia characterised by the presence of the PML-RARA fusion transcript. The present standard of care, chemotherapy and all-trans retinoic acid (ATRA), results in a high proportion of patients being cured. In this study, we compare a chemotherapy-free ATRA and arsenic trioxide treatment regimen with the standard chemotherapy-based regimen (ATRA and idarubicin) in both high-risk and low-risk patients with acute promyelocytic leukaemia. Methods: In the randomised, controlled, multicentre, AML17 trial, eligible patients (aged ≥16 years) with acute promyelocytic leukaemia, confirmed by the presence of the PML-RARA transcript and without significant cardiac or pulmonary comorbidities or active malignancy, and who were not pregnant or breastfeeding, were enrolled from 81 UK hospitals and randomised 1:1 to receive treatment with ATRA and arsenic trioxide or ATRA and idarubicin. ATRA was given to participants in both groups in a daily divided oral dose of 45 mg/m(2) until remission, or until day 60, and then in a 2 weeks on-2 weeks off schedule. In the ATRA and idarubicin group, idarubicin was given intravenously at 12 mg/m(2) on days 2, 4, 6, and 8 of course 1, and then at 5 mg/m(2) on days 1-4 of course 2; mitoxantrone at 10 mg/m(2) on days 1-4 of course 3, and idarubicin at 12 mg/m(2) on day 1 of the final (fourth) course. In the ATRA and arsenic trioxide group, arsenic trioxide was given intravenously at 0·3 mg/kg on days 1-5 of each course, and at 0·25 mg/kg twice weekly in weeks 2-8 of course 1 and weeks 2-4 of courses 2-5. High-risk patients (those presenting with a white blood cell count >10 × 10(9) cells per L) could receive an initial dose of the immunoconjugate gemtuzumab ozogamicin (6 mg/m(2) intravenously). Neither maintenance treatment nor CNS prophylaxis was given to patients in either group. All patients were monitored by real-time quantitative PCR. Allocation was by central computer minimisation, stratified by age, performance status, and de-novo versus secondary disease. The primary endpoint was quality of life on the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 global health status. All analyses are by intention to treat. This trial is registered with the ISRCTN registry, number ISRCTN55675535. Findings: Between May 8, 2009, and Oct 3, 2013, 235 patients were enrolled and randomly assigned to ATRA and idarubicin (n=119) or ATRA and arsenic trioxide (n=116). Participants had a median age of 47 years (range 16-77; IQR 33-58) and included 57 high-risk patients. Quality of life did not differ significantly between the treatment groups (EORTC QLQ-C30 global functioning effect size 2·17 [95% CI -2·79 to 7·12; p=0·39]). Overall, 57 patients in the ATRA and idarubicin group and 40 patients in the ATRA and arsenic trioxide group reported grade 3-4 toxicities. After course 1 of treatment, grade 3-4 alopecia was reported in 23 (23%) of 98 patients in the ATRA and idarubicin group versus 5 (5%) of 95 in the ATRA and arsenic trioxide group, raised liver alanine transaminase in 11 (10%) of 108 versus 27 (25%) of 109, oral toxicity in 22 (19%) of 115 versus one (1%) of 109. After course 2 of treatment, grade 3-4 alopecia was reported in 25 (28%) of 89 patients in the ATRA and idarubicin group versus 2 (3%) of 77 in the ATRA and arsenic trioxide group; no other toxicities reached the 10% level. Patients in the ATRA and arsenic trioxide group had significantly less requirement for most aspects of supportive care than did those in the ATRA and idarubicin group. Interpretation: ATRA and arsenic trioxide is a feasible treatment in low-risk and high-risk patients with acute promyelocytic leukaemia, with a high cure rate and less relapse than, and survival not different to, ATRA and idarubicin, with a low incidence of liver toxicity. However, no improvement in quality of life was seen.
      pubtype: Academic Journal
      doctype:
        research
        randomized controlled trial
        Journal Article
      ougenre: Article
    language: English
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