RESUMEN
Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS) is a surrogate marker for symptom evaluation in Chronic Myeloproliferative Neoplasms. There is not enough data to show the relationship between MPN-SAF TSS, JAK2 mutation allele burden, and thrombosis. In this retrospective analysis, we aimed to determine the genetic burdens, clinical features, and relationship with MPN-SAF TSS in MPN patients. One hundred thirty JAK2V617F positive MPN were included in our study. We have calculated MPN-SAF TSS and compared it with clinical features. Patients with higher JAK2V617F mutation allele burden had higher MPN-SAF TSS (p-value 0,008). Patients with thrombosis had higher MPN-SAF TSS scores than patients without thrombosis (p-value 0.003). The mean MPN-SAF TSS was higher in primary myelofibrosis (PMF) patients compared to PV and ET patients. Thrombosis was associated with increased symptom severity in several domains, including fatigue, abdominal discomfort, inactivity, night sweats, pruritus, weight loss, and early satiety. Additionally, an increase in JAK2 allele burden was observed with higher symptom scores. The MPN-SAF TSS proved to be a reliable tool for assessing symptom burden in Turkish MPN patients. Furthermore, a significant association between thrombosis occurrence and symptom severity suggests that thrombotic events may contribute to symptom development. Notably, increasing JAK2 allele burden was correlated with more severe symptoms, highlighting its potential role in predicting disease burden. This study emphasizes the importance of symptom assessment in MPN patients and supports the incorporation of MPN-SAF TSS in routine clinical practice to enhance patient care and management.
RESUMEN
Introduction: Primary immune thrombocytopenia (ITP) is an acquired disorder of platelets with complex and unclear mechanism of increased immune distruction or impaired production of platelets. While management of ITP is evolving, there is a need for guidance particularly in certain circumstances such as pregnancy, emergency and for patients requiring co-medications. We aimed to determine the tendencies of hematologists in Turkiye on such special conditions. Methods: As a modified Delphi method, Turkish National ITP Working Group founded under Turkish Society of Hematology developed a questionnaire consisting of statements regarding pregnancy, emergency and circumstances regarding co-treatment with antiaggregant or anticoagulants. 107 Hematologists working either in university or state hospitals voted for their agreement or disagreement of the statements for two consequential rounds. Results: Participant hematologists reached an agreement on the starting treatment in pregnant patients with platelets less than 30 x109/L and delivery of either normal or cesarian section to be safely performed above 50 x109/L. For emergency and rescue management of ITP, our panel have agreed against the use of high dose corticosteroids alone, preferred a combination with transfusion or IVIG. For patients who require interventions, platelet counts >50 x109/L were regarded as safe for low risk procedures as well as co-treatment with antiplatelets or anticoagulants. Conclusion: As National ITP study group, we have observed the need to increase the practice guidance in patients with primary ITP requiring additional treatments including invasive interventions, and co-treatments towards coagulation. Decisions on the management of ITP during pregnancy should be individualized. There is a certain lack of consensus on the thresholds of platelet counts as well as co-morbidities and co-medications. This lack of consensus may be due to the variations in the practices.
RESUMEN
Objective: Primary immune thrombocytopenia (pITP) is an acquired autoimmune disorder related to the increased destruction and/or impaired production of platelets. Its diagnosis and management are challenging and require expertise and the interpretation of international consensus reports and guidelines with national variations in availability. We aimed to assess the agreement of hematologists in Türkiye on certain aspects of both first-line and second-line management of patients with pITP. Materials and Methods: Applying a modified Delphi method, the Turkish National ITP Working Group (14 steering committee members), founded under the auspices of the Turkish Society of Hematology, developed a 21-item questionnaire consisting of statements regarding the first-line and second-line treatment of pITP. A total of 107 adult hematologists working in either university or state hospitals voted for their agreement or disagreement with the statements in two consecutive rounds. Results: The participants reached consensus on the use of corticosteroids as first-line treatment and with limited duration. Methylprednisolone was the corticosteroid of choice rather than dexamethasone. Use of intravenous immunoglobulin was not preferred for patients without bleeding. It was also agreed that thrombopoietin receptor antagonists (TPO-RAs) or rituximab should be recommended as second-line treatment and that splenectomy could be considered 12-24 months after diagnosis in patients with chronic pITP. Conclusion: The optimization of the dose and duration of TPO-RAs in addition to corticosteroids is necessary to improve the management of patients with pITP.