INTRODUCTION: The actual scenario of patients treated with acute lymphoblastic leukemia (ALL) in Brazil is not well understood. The literature addressing patients reported outcomes (PRO) about diagnosis, treatment and adverse effects (AE) is lacking. OBJECTIVE: To evaluate patients' reported experience about ALL and to investigate associations between PRO, AE, demographics, healthcare system and disease information. PATIENTS AND METHODS: A prospective cross-sectional study interviewed by telephone ALL patients/caregivers registered in the Brazilian Lymphoma and Leukemia Association (ABRALE). RESULTS: 400 patients treated between 1981-2019, 203 male, mean age 15.7 years (range:0-77), median follow-up 6.2 years (range:0.9-38.6); most reported presentation symptoms were: fever (39%), bleeding/ecchymosis (38%), intense fatigue (30%), musculoskeletal pain (28%); the proportion of patients diagnosed within ≤ 1week since symptoms differed between public vs private healthcare: 17.9% vs 31.1% (p0.019); more patients reported diagnostic difficulties on public care: 35% vs 22.6% (p0.034); 80/134(59.8%) reported multiple referrals/medical visits until diagnosis; 24% reported information of minimal residual disease (MRD) status: MRDpos 22(23.9%) and MRDneg 77(76.1%); only 9% could inform treatment protocol; from 8 reported protocols, GBTLI 10(27.8%) and BFM 8(22.2%) were most frequent; 12% patients reported difficulties to start treatment, mainly related to dug unavailability (54%) or own expenditure to drug access (25%). 17.5% reported treatment modification: 35(50%) due to refractory/relapse; 26(37.1%) related to severe AE; 206(51.5%) reported treatment duration ≥2years and 27.2% reported cranial radiotherapy (cRT); 91(22.7%) reported allo-HSCT indication, among 64(70.3%) whose had undergone it, 17(27%) reported difficulties on allo-HSCT step, mainly related to >3months delay to find donor. Factors associated with allo-HSCT indication were: MRDpos (OR:3.22 95%CI:1.07-9.66 p0.037), cRT (OR:4.36 95%CI:1.61-11.8 p0.004); 167(41.7%) reported AE attributed to treatment, median 1 AE/patient (range:1-6); Frequently reported AE were: mood disorders (44/26.3%), neurologic deficit (23/13.8%), cognitive/memory (20/12%), lung disease (25/15%); Risk factors to experiencing AE were: age (OR:1.019/year 95%CI:1.00-1.03 p0.003); Female gender (OR:1.52 95%CI:0.99-2.31 p0.05); allo-HSCT indication (OR:1.67 95%CI:1.004-2.78 p0.048); to live in capital city (OR:1.92 95%CI:1.26-2.92 p0.002). CONCLUSIONS: In this study patients remain facing challenges, such as diagnostic and allo-HSCT delay, drug unavailability and poor disease information. Indeed, there is large treatment heterogeneity and different durations, represented by 8 different protocols in a small patient subset and high rates of cRT. 41.7% patients reported AE, mainly affecting neuropsychological and lung. Finally, there is an urgent call to define national ALL referral centers and diagnostic/treatment standards to improve patients care.
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Disclosures
No relevant conflicts of interest to declare.
