An audit of platelet transfusion indications in acute leukaemia patients: six-year experience at an Academic Centre.

BACKGROUND: Platelet transfusion plays a critical role in the supportive treatment of acute leukaemia patients who receive chemotherapy and haematopoietic stem cell transplantation (HSCT). There are few studies assessing appropriateness of platelet transfusion in this population. An audit was conducted to determine how appropriately platelets are transfused in acute leukaemia patients at a tertiary care health institution. MATERIALS AND METHODS: A six-year retrospective audit was conducted in acute lymphoblastic (ALL) and acute myeloid leukaemia (AML) patients in an Academic Centre. Episodes were assessed as either appropriate or inappropriate based on guidelines from the British Society for Haematology (BSH). Pre-transfusion platelet count, transfusion indication, World Health Organization (WHO) bleeding score, and antibiotic use were all documented. RESULTS: Overall, 745 platelet transfusion episodes in 154 patients were audited. The proportion of episodes appropriately indicated according to BSH guidelines was 75.3%. Paediatrics and Internal Medicine had the lowest and highest proportion of appropriateness by department at 63.9% and 86.8%, respectively. The best alignment to guidelines was found on the wards (82.3%). Inpatient cases were significantly better indicated (p=0.002), whereas therapeutic and HSCT-related transfusions were not. The majority of inappropriate transfusions had a pre-transfusion count >20×109/L without a valid justification (45.1%), whereas appropriate episodes were mainly accounted for by a pre-transfusion count <10×109/L (69%). DISCUSSION: The 25% rate of inappropriate platelet transfusion in acute leukaemia patients underscores the learning needs of physicians, particularly those in training, regarding adequate use of platelets in haematologic malignancies to optimise its utilisation and patient outcome.

Advances in gene therapy for hemophilia.

Hemophilia is a hereditary disorder that can be life-threatening in individuals who have severe spontaneous bleeding resulting from minor trauma or surgery. Although replacement therapy of the missing exogenous factor has improved patients' quality of life, it has not been possible to establish a long-term treatment. Due to the severity of the disease and the need for repetitive doses throughout the patient's life, replacement therapy has become a high-cost treatment option; therefore, the development of self-sustainable long-term therapies is critical. Hemophilia is a good candidate for gene therapy because it is a monogenic disease that can be counteracted by expression of the missing factor. In this article, we review some of the most relevant advances in gene therapy for this illness.

Real-world outcomes of treatment for acute lymphoblastic leukemia during adolescence in a financially restricted environment: Results at a single center in Latin America.

OBJECTIVE: There is a paucity of the studies of adolescents with acute lymphoblastic leukemia (ALL). This is more noticeable in low- and middle-income countries. The international 5-year event-free survival (EFS) and overall survival (OS) for this age group is around 80%, with pediatric-inspired protocols offering better results. METHODS: A retrospective analysis of adolescents aged 16-20 diagnosed with ALL during the period 2004-2015 treated with a high-risk pediatric protocol at an academic center from a middle-income country was performed. Five-year OS and EFS were estimated by the Kaplan-Meier analysis. Hazard ratios of relapse and death were estimated by the Cox regression model. RESULTS: Five-year EFS and OS for 57 adolescents were 23.3% and 48.9%, respectively. From the 41 patients who achieved complete remission, 24 (58.5%) relapsed. Bone marrow and central nervous system were the most frequent sites of relapse. Hazard ratio of treatment failure and death for patients with organomegaly at diagnosis was 2.026 and 2.970, respectively. Treatment-related toxicity developed in 31 (54.4%) patients and febrile neutropenia was the most frequent in 14 (24.6%) cases. Twelve patients (21.1%) had poor adherence to treatment. CONCLUSIONS: High relapse rate and low 5-year EFS compared with international standards, was documented. Use of intensified pediatric regimens, adherence to proven effective medications, improved supportive care, and prevention of abandonment are necessary to improve survival rates in these patients.