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[Symptomatic Profiles of 173 Chinese Patients with Essential Thrombocythemia]
Ting Pan1, Xiao-Fan Liu1, Wei Liu1
1State Key Laboratory of Experimental Hematology, Key Laboratory of Gene Therapy for Blood Disease, Institute of Hematology and Blood Disease Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, 300020, China.
Insights
Essential thrombocythemia (ET) patients experience varying symptom burdens influenced by clinical factors. Hydroxyurea treatment, JAK2V617F mutation, and female sex are associated with higher symptom burden in ET.
Area of Science:
- Hematology
- Oncology
- Clinical Medicine
Background:
- Essential thrombocythemia (ET) is a myeloproliferative neoplasm characterized by elevated platelet counts.
- Understanding the symptomatic burden in ET patients is crucial for effective management and treatment strategies.
Purpose of the Study:
- To investigate the symptom burden in Chinese ET patients.
- To analyze the relationship between symptom burden and clinical characteristics such as mutation status, therapeutic protocols, and sex.
Main Methods:
- A cohort of 173 Chinese ET patients was analyzed.
- Patients were categorized based on disease characteristics including JAK2V617F mutation, CALR mutation, hydroxyurea (Hu) treatment, interferon-alpha (IFN-α) treatment, and sex.
Main Results:
- Symptom burden varied across groups, with the highest Total Symptom Score (TSS) observed in the hydroxyurea-treated group.
- JAK2V617F-positive, hydroxyurea-treated, and female patients reported higher TSS and individual symptom scores.
- CALR-positive and IFN-α-treated groups exhibited lower symptom scores compared to their counterparts.
- An increasing number of risk factors (JAK2V617F-positive, Hu-treated, female) correlated with a greater symptom burden.
Conclusions:
- Clinical characteristics significantly influence the symptom burden experienced by ET patients.
- The cumulative effect of specific characteristics exacerbates symptom severity.
- Comprehensive consideration of patient characteristics is essential for tailoring treatment plans and predicting prognosis in ET.
Objective:
To explore the symptomatic burden of patients with essential thrombocythemia (ET) and its relation with clinical characteristics including the mutation status, therapeutic protocols and sex.
Methods:
Total of 173 Chinese ET patients were selected and grouped on the basis of disease characteristics (mutation status, therapeutic pro to- cols, and sex).
Results:
All the groups showed low-to-high symptom burden, with the highest in the Hu (hydroxyurea)-group (total symptom score [TSS], 14.7; range, 7.6-14.7). In the JAK2V617F-positive, Hu-treated, and female groups TSS and independent symptom scores were higher than those in the control group. The CALR-positive and IFN-α-treated groups had lower overall and individual scores as compared with groups lacking the corresponding characteristics. As the number of characteristics (JAK2V617F-positive, Hu-treated, and female) increases, the severity of symptoms gradually increased.
Conclusion:
The different characteristics have various effects on symptom burden in ET patients. The accumulation of certain characteristics will lead to more severe symptom burden, thus the patient's symptom burden should be considered comprehensively when making up the treatment schemes and prognosis.
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