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Maria Mareque,1 Pau Montesinos,2 Patricia Font,3 José María Guinea,4 Adolfo de la Fuente,5 Javier Soto,6 Itziar Oyagüez,1 James Brockbank,7 Tamara Iglesias,6 Julia Llinares,6 Jorge Sierra8 1Pharmacoeconomics & Outcomes Research Iberia (PORIB), Madrid, Spain; 2Hospital Universitario La Fe, Valencia, Spain; 3Hospital General Universitario Gregorio Marañón, Madrid, Spain; 4Organización Sanitaria Integrada Araba, País Vasco, Spain; 5Hospital MD Anderson, Madrid, Spain; 6Pfizer, Madrid, Spain; 7RTI Health Solutions, Manchester, UK; 8Hospital Universitario de La Santa Creu i Sant Pau, Barcelona, SpainCorrespondence: Maria MarequePharmacoeconomics & Outcomes Research Iberia (PORIB), Paseo Joaquín Rodrigo 4-I, Pozuelo de Alarcón, Madrid, 28224, SpainTel + 34 91 715 91 47Fax + 34 91 715 94 69Email mmareque@porib.comObjective: To assess the incremental cost–utility ratio (ICUR) of gemtuzumab ozogamicin (GO) + standard of care (SOC) vs SOC alone for treatment of patients with de novo AML from a Spanish Health Service perspective.Methods: A cohort state-transition model, with 12 health-states, was used to estimate the lifetime accumulated cost and benefits in terms of quality-adjusted-life-years (QALYs) in AML patients with favourable, intermediate, and unknown cytogenetic profiles. Patient profile was defined based on the ALFA-0701 trial. Therapeutic regimens were defined by 5 haematologists. SOC was assumed to be idarubicin and cytarabine, the combination most used in Spain. QALYs were estimated by applying utilities for the time spent by the cohort in each health-state and utility decrements associated with adverse events (AE). Total cost (€,2020) included drug-acquisition, hematologic stem-cell transplantation, disease management, AE management and end-of-life costs. Unit costs were derived from local databases. All parameters were validated by haematologist. Costs and outcomes were discounted (3%/year).Results: Higher cost/patient (€ 177,618 vs € 151,434) and greater QALYs (5,70 vs 4,62) were obtained with GO+SOC vs SOC. The ICUR was € 24,203/QALY gained.Conclusion: This simulation suggests that GO + SOC could be a cost-effective option for treatment of patients with de novo AML in first line.Keywords: cost-effectiveness, gemtuzumab ozogamicin, acute myeloid leukaemia, Spain

The use of non-pegylated liposomal doxorubicin (Myocet ® ) in diffuse large B-cell lymphoma (DLBCL) has been investigated in retrospective and single-arm prospective studies. This was a prospective phase 2 trial of DLBCL patients ≥60 years old with left ventricular ejection fraction (LVEF) ≥55% randomized to standard R-CHOP or investigational R-COMP (with Myocet ® instead of conventional doxorubicin). The primary end point was to evaluate the differences in subclinical cardiotoxicity, defined as decrease in LVEF to <55% at the end of treatment. Secondary objectives were efficacy, safety, and variations of troponin and N-terminal pro-B-type natriuretic peptide (NT-proBNP) and LVEF along follow-up. Ninety patients were included, 45 in each group. No differences were observed in the percentage of patients with LVEF <55% at end of treatment (11% in R-CHOP arm vs. 7% in R-COMP arm, p = 0.697) or at 4 months (10% vs. 6%, respectively, p = 0.667) and 12 months (8% vs. 7%, respectively, p = 1). However, a higher percentage of R-CHOP compared with R-COMP patients showed increased troponin levels in cycle 6 (100% vs. 63%, p = 0.001) and at 1 month after treatment (88% vs. 56%, respectively, p = 0.015). Cardiovascular adverse events were seen in five R-CHOP patients (nine episodes, four grade ≥3) and in four R-COMP patients (five episodes, all grade 1-2). No significant differences in efficacy were observed. In conclusion, R-COMP is a feasible immunochemotherapy schedule for DLBCL patients ≥60 years, with similar efficacy to R-CHOP. However, the use of non-pegylated doxorubicin instead of conventional doxorubicin was not associated with less early cardiotoxicity, although some reduced cardiac safety signals were observed. Trial registration: ClinicalTrials.gov Identifier: NCT02012088., (© 2021 The Authors. Cancer Medicine published by John Wiley & Sons Ltd.)

The role of bosutinib as rescue treatment of Philadelphia chromosome-positive chronic myeloid leukemia (CML) patients after failing three previous tyrosine kinase inhibitors (TKIs) is currently unknown. We report here the largest series (to our knowledge) of patients treated with bosutinib in fourth-line, after retrospectively reviewing 30 patients in chronic phase, and pretreated with imatinib, nilotinib, and dasatinib. With a median follow up of 11.1 months, the probability to either maintain or improve their CCyR response was 56.6% (17/30) and 11 patients (36.7%) achieved or maintained their baseline MMR. In patients not having baseline CCyR, the probabilities of obtaining CCyR, MMR, and MR4.5 were 13, 11, and 14%, respectively. The probabilities of obtaining MMR and deep molecular response MR4.5 in patients with baseline CCyR were 40.0% (6/15) and 20.0% (3/15). At 20 months, progression-free survival was 73%. Grade 3-4 hematological toxicities were more frequent in resistant than intolerant patients (45.4 vs. 0.0%). Nonhematological toxicities were also more frequent in resistant patients, being diarrhea the most conspicuous one. Bosutinib seems to be an appropriate treatment option for patients resistant or intolerant to three prior TKI's., (© 2015 Wiley Periodicals, Inc.)

Purpose: The optimal postremission therapy for children with very high-risk (VHR) acute lymphoblastic leukemia (ALL) is not well established. This randomized trial compared three options of postremission therapy: chemotherapy and allogeneic or autologous stem-cell transplantation (SCT)., Patients and Methods: All 106 VHR-ALL patients received induction with five drugs followed by intensification with three cycles of chemotherapy. Patients in complete remission (CR) with an HLA-identical family donor were assigned to allogeneic SCT (n = 24) and the remaining were randomly assigned to autologous SCT (n = 38) or to delayed intensification followed by maintenance chemotherapy up to 2 years in CR (n = 38)., Results: Overall, 100 patients achieved CR (94%). With a median follow-up of 6.5 years, 5-year disease-free survival (DFS) and overall survival (OS) probabilities were 45% (95% CI, 37% to 54%) and 48% (95% CI, 40% to 57%), respectively. The three groups were comparable in the main pretreatment ALL characteristics. Intention-to-treat analysis showed no differences for donor versus no donor in DFS (45%; 95% CI, 27% to 65% v 45%; 95% CI, 37% to 55%) and OS (48%; 95% CI, 30% to 67% v 51%; 95% CI, 43% to 61%), as well as for autologous SCT versus chemotherapy comparisons (DFS: 44%; 95% CI, 29% to 60% v 46%; 95% CI, 32% to 62%; OS: 45%; 95% CI, 31% to 62% v 57%; 95% CI, 43% to 73%). No differences were found within the different subgroups of ALL and neither were differences observed when the analysis was made by treatment actually performed., Conclusion: This study failed to prove that, when a family donor is available, allogeneic SCT produces a better outcome than autologous SCT or chemotherapy in children with VHR-ALL.

Purpose: Restrictive erythropoiesis caused by iron deficiency may hinder pre-deposit autotransfusion in surgical procedures. In order to evaluate the response to prophylactic ferrous ascorbate, a prospective study was conducted on patients subjected to orthopaedic surgery and autotransfusion., Patients and Methods: Sixty-eight patients were included in the study: hip prostheses 67%, knee prostheses 25%, other procedures 7.4%. Their mean age was 61.3 +/- 10.2 years, and there were 42% male and 57% female. A mean of 2.8 +/- 0.6 units (450 mL) of blood were drawn to each patient in a month. Starting one week before their first blood donation and up to 2 months after surgery, each patient received 99 mg elementary iron per days as oral ferrous ascorbate. Blood cell counts were done at the beginning of the programme and after the first, second, third and fourth blood withdrawal, as well as one month after finishing the treatment. A survey of iron profile including serum iron, total iron binding capacity, transferrin saturation, serum ferritin and free erythrocyte protoporphyrin was carried out at onset and end of the programme in each patient. All data were analysed with the SPSS-PC 4.0 statistical programme., Results: Haemoglobin rates decreased in every control, returning to values close to the initial ones by the end of the programme (mean figures are as follows: 14.63; 13.17; 12.70; 11.88; 14.11 g/dL); and similar changes were seen with respect to the other parameters of blood. The initial and final values for ferritin were 157.32 and 91.06 ng/mL, respectively, and no significant changes were appreciated in the other data from the iron profile, regardless of the number of blood units collected in a given case. Minor intolerance to ferrous ascorbate appeared in 11% of the patients. No significant differences with control patients were seen regarding hospitalization (16.54 vs 19.82 days) or postoperative fever (14.1% vs 17.11%)., Conclusions: As opposed to others, we feel that iron treatment should be maintained up to 2 months after surgery since better results are thus attained. Recombinant erythropoietin is more expensive a method. Ferrous ascorbate is better tolerated than ferrous sulphate plus additives.