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The content of this article is only for medical and health professionals.
ITP disease background and treatment progress Primary immune thrombocytopenia (ITP) is a bleeding disorder caused by autoimmune dysfunction in which the number of platelets is reduced
.
Worldwide, the incidence of ITP is 2-10/100,000, and the peak incidence is in people aged 20-30 and over 60 years old 1,2
.
The clinical goal of ITP therapy is to resolve bleeding events or prevent severe bleeding by providing adequate hemostasis
.
At present, the first-line treatment drugs are mainly glucocorticoids, but there are some cases where hormone therapy is ineffective in some patients
.
Hormone ineffective means that platelet count is less than 30×109/L after hormone therapy, or platelet count is less than 2 times of the basal value, or there is bleeding
.
The results of the pooled analysis showed that approximately 30% of patients did not achieve initial response to hormone therapy 4
.
The "Chinese Guidelines for the Diagnosis and Treatment of Primary Immune Thrombocytopenia in Adults (2020 Edition)" pointed out that patients who are ineffective or relapsed after high-dose dexamethasone treatment can be repeated for 1 cycle (1 cycle 4d); prednisone treatment within 2 weeks Ineffective patients should stop as soon as possible1
.
Let's hear what the experts have to say about the goals of treatment for ITP patients, the unmet needs of first-line hormone therapy, and the options for follow-up clinical treatment when hormone therapy is ineffective
.
Expert concept and clinical practice sharing Experts in this issue: Prof.
Dong Ruihong, Prof.
Hu Qian, Prof.
Zhou Xiaofen, Prof.
Song Bin Prof.
Dong Ruihong, Attending Physician of Department of Hematology, Shunde Hospital, Southern Medical University, Member of Coagulation Group of Guangdong Medical Doctor Association Member of the Chronic Myeloid Leukemia/Myeloproliferative Tumor Working Group Member of the 1st Hematological Cancer Prevention and Treatment Professional Committee of Guangdong Preventive Medicine Association Expert Concepts What factors should be considered in the subsequent selection of treatment options for patients with poor first-line hormone control, Dong Ruihong The professor believes: "For patients with poor first-line hormone control, when choosing a treatment plan, consider entering second-line treatment, and if the patient has sufficient economic conditions, thrombopoietin receptor agonists (TPO-RA) will be preferred, such as Eltrombopag is used at a full dose of 75 mg.
If economic conditions do not allow it, try to slowly reduce the dose of hormones and combine with low-dose TPO-RA, and observe whether the platelet count changes and whether the bleeding symptoms are relieved
.
"Clinical practice sharing Case 1 Basic information: Gender: Female Age: 82 years old Initial platelet level: 5×109/L Hormone ineffective: Hormone ineffective Table 1 Case 1 Follow-up and efficacy evaluation records Note: Hormones and eltrombopag are administered daily once (qd)
.
Figure 1 The follow-up and efficacy evaluation of case 1 recorded that the initial platelet count of the patient was 5×109/L, and after 9 weeks of eltrombopag (50mg, qd) treatment, the platelet count increased to 68×109/L, reaching the therapeutic level Effective, that is, the platelet count after treatment is ≥30×109/L, which is at least 2 times higher than the basic platelet count, and there is no bleeding
.
Professor Hu Qian, Chief Physician of the Department of Hematology, Chengdu First People's Hospital, Member of the Sichuan Geriatric Hematology Professional Committee, Member of the Chengdu Hematology Professional Committee What are the unmet needs of hormone therapy, Professor Hu Qian believes: "The use of hormone therapy in ITP patients has serious side effects, and long-term use of hormones will lead to poor control
.
"Clinical practice sharing Case 2 Basic information: Gender: Female Age: 52 years old Initial platelet level: 18 × 109/L Hormone incompetence: Hormone ineffective Table 2 Case 2 Follow-up and efficacy evaluation records Note: Hormone and Eltrombopag medication All were once a day (qd)
.
Figure 2 The follow-up and efficacy evaluation of case 2 recorded that the patient's initial platelet count was 18 × 109/L, and after 9 weeks of eltrombopag (50 mg, qd) treatment, the platelet count increased To 190×109/L, a complete response is achieved, that is, the platelet count is ≥100×109/L, and there is no bleeding
.
Professor Zhou Xiaofen, Deputy Chief Physician of Xiangyang Central Hospital, Master of Medicine Member of the Society of Hematology Graduated from Tongji Medical College of Huazhong University of Science and Technology in 2008 and obtained a master's degree in medicine from the clinical and teaching work of Xiangyang Central Hospital.
He has published a number of SCI expert concepts on the treatment goals of ITP patients.
Rapidly increase the patient's platelet count to a safe level (≥30×109/L); stabilize the platelet count and reduce the risk of bleeding; improve the patient's quality of life
.
"Clinical practice sharing Case 3 Basic information: Gender: Female Age: 46 years old Initial platelet level: 4 × 109/L Hormone ineffectiveness: Hormone ineffective Table 3 Case 3 Follow-up and efficacy evaluation records Note: Hormone and Eltrombopag medication All were once a day (qd).
Figure
3
The follow-up and efficacy evaluation of case 3 recorded that the patient's initial platelet count was 4 × 109/L, and after 9 weeks of eltrombopag (50mg, qd) treatment, the platelet count increased To 85×109/L, the treatment is effective, that is, the platelet count after treatment is ≥30×109/L, which is at least 2 times higher than the basic platelet count, and there is no bleeding
.
Professor Song Bin, Deputy Director of the Department of Hematology, Shiyan Taihe Hospital, Master Supervisor Published more than 20 papers in core journals, hosted or participated in a number of national and provincial scientific research projects, edited 1 monograph, and patented 2 expert concepts for the treatment goals of ITP patients.
, to minimize the patient's bleeding symptoms; the patient's platelets can be stable for a long time after the rapid increase; the patient has no obvious side effects during the treatment process, and the quality of life is high; the treatment drugs can be reduced and stopped
.
"Clinical practice sharing case 4 Basic information: gender: female Age: 63 years old Initial platelet level: 31×109/L Hormone inadequacy: Hormone ineffective Table 4 Case 4 Follow-up and efficacy evaluation records Note: Hormones and Eltrombopag are administered once a day (qd)
.
Figure 4 The follow-up and efficacy evaluation of case 4 recorded that the initial platelet count of the patient was 31×109/L, and after 9 weeks of eltrombopag (75mg, qd) treatment, the platelet count increased to 175×109/L, reaching complete Response, that is, platelet count ≥ 100 × 109/L, and no bleeding manifestations
.
Reference 1.
Hou Ming, Hu Yu.
Chinese Journal of Hematology, 2020(8):617-623.
2.
Frederiksen, H.
et al.
Blood.
1999;94:909-913.
3.
Provan, D.
et al.
Blood Adv 2019;3:3780-3817.
4.
Cuker A, et al.
Res Pract Thromb Haemost.
2021;5(6):e12592.
MCC No.
REV22041922 is valid on 2023-04-18, and the data is expired and will be deemed invalid
.
Edit: September Typography: Quinta Execution: Wenting pokes "read the original", we progress together