RESUMO
Chronic myeloid leukemia (CML) is a myeloproliferative disorder due to translocation between chromosomes (9, 22), known as the "Philadelphia chromosome." In 2016, the World health organization (WHO) introduced a new clinical entity of de novo acute myeloid leukemia (AML). Both diseases share some commonalities, therefore, create a challenge to diagnose.
RESUMO
BACKGROUND: Eltrombopag olamine (ELT) is a synthetic nonpeptide with a low molecular weight that has been investigated in various phase-3 studies and shown to be efficacious at a typical dose of 50 mg. Varied ethnic groups have reported different responses to ELT. AIM: The aim is to examine the efficacy of ELT in Asian and Arab patients with immune thrombocytopenia (ITP) from the Indian subcontinent by starting with (12.5 mg, as a minimum dose) and gradually increasing to a maximum dose of 50 mg. METHODS: Between January 2015 and January 2019, we reviewed the electronic health records of non-Arab Asian (n = 17) versus Arab (n = 41) patients who were ≥18 years old, residing in Qatar, and with confirmed diagnoses with chronic ITP and under active treatment with a platelet count of 30,000/L, and bleeding symptoms. Following receiving ELT for three months or longer at various dosages, patients' response was examined. RESULTS: After three months of ELT therapy, the response rate (platelet count of 50,000/L) was equivalent in non-Arab (88.2%) versus Arab (87.5%) patients. However, to achieve an adequate response, 26% of Arab patients required a lower dose of 12.5 or 25 mg, and 41.5% required a higher dose of 50 mg. CONCLUSION: In adult chronic ITP patients, ELT is typically well-tolerated and delivers the desired outcomes. In 67.5% of Arab patients, smaller dosages of ELT (12.5-50 mg) were helpful in sustaining acceptable PLT levels. This helps patients get the most benefit at the lowest feasible dose, reducing toxicity and expense.
RESUMO
Essential thrombocythemia (ET) is one of the myeloproliferative neoplasms, characterized by persistent thrombocytosis, platelets >450,000/µL, and evident clonal abnormalities like JAK2 V617F, MPL, CALR mutation and not fulfilling WHO criteria for MDS, CML, PV, and IDA. Here we report a 24-year-old female who presented with headache and was found to have thrombocytosis with a platelet count of 2,141 × 103/µL, diagnosed as ET as per WHO criteria 2008; she required ICU admission and thrombocytapheresis with a favorable outcome.
RESUMO
Despite regular blood transfusion and iron chelation therapy, growth impairment and pubertal delay are commonly seen in children and adolescents with transfusion-dependent Beta thalassaemia major (BTM) and sickle cell disease (SCD). We evaluated growth parameters and endocrine disorders in relation to the liver iron concentration (LIC) assessed by the Ferriscan® method in a cohort of adults with SCD (n =40) and BTM (n = 52) receiving blood transfusions and iron chelation therapy since early childhood. Before transfusion, hemoglobin concentration had not been less than 9 g/dl in the past 12 years; subcutaneous daily desferrioxamine was administered for all of them since early childhood (2- 5 years of age). All patients were shifted to oral therapy with deferasirox iron chelation, 20 mg/daily for the past 5 years. BTM patients with higher LIC (> 15 mg Fe/g dry weight) had significantly shorter stature, lower insulin-like growth factor-I SDS (IGF-I SDS), higher alanine transferase (ALT) and serum ferritin concentrations compared to thalassemic patients with lower LIC. Patients with SCD with LIC > 8 mg Fe/g dry weight had significantly shorter stature, lower IGF-I SDS and higher ALT compared to SCD patients with lower LIC. Patients with BTM had significantly shorted final height (Ht-SDS) , IGF-I SDS and FT4 level compared to patients with SCD. LIC and mean fasting blood glucose (FBG) were significantly higher in patients with BTM compared to those with SCD. The linear regression analysis showed a significant correlation between LIC and serum ferritin level in SCD and BTM. LIC and serum ferritin level were also correlated significantly with IGF-I level in patients with BTM. LIC was correlated significantly with ALT in patients with BTM. In conclusion, the prevalence of endocrinopathies especially hypothyroidism, DM, and hypogonadism were significantly higher in BTM patients versus SCD patients and higher in patients with higher LIC versus those with lower LIC. These complications occurred less frequently, but still considerable, in chronically transfused patients with SCD.
