Call to Include ITP in Outpatient Reimbursement to Alleviate Long-Term Treatment Burden

Beijing, May 25, 2020 /PRNewswire/ -- On May 24, 2020, the "China Hematology Summit," hosted by Novartis Oncology, was convened. At this summit, leading experts from across China in the fields of clinical practice and research in hematologic oncology shared and discussed the latest therapeutic advances and diagnostic and treatment experiences in hematologic diseases via a special online video format.

“Among the numerous blood disorders defined by the World Health Organization (WHO), many are often overlooked due to limited public awareness, in contrast to malignant conditions such as leukemia and lymphoma. Primary immune thrombocytopenia (ITP) is one such disorder,” emphasized Professor Yang Renchi from the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, while sharing updates on the diagnosis and treatment of ITP. “In fact, ITP is the most common hemorrhagic disorder encountered in clinical practice. Under previous treatment paradigms, patients with this condition experienced poor prognoses and a substantial burden, with quality of life even lower than that of patients with chronic diseases such as cancer, hypertension, and diabetes.”

ITP: A Potentially Fatal “Invisible Killer”

ITP, also known as idiopathic thrombocytopenic purpura, is a common acquired autoimmune hemorrhagic disease, accounting for approximately one-third of all hemorrhagic diseases. According to statistics, the incidence of ITP in the adult population in China is 5–10 per 100,000.^[1]More common in the elderly aged over 60, women of childbearing age, and children.^[2]。

The etiology of ITP is primarily attributed to immune dysfunction, which leads to increased destruction and decreased production of platelets—the body's "clotting guardians." Consequently, platelet counts fail to reach the normal physiological range, resulting in impaired blood coagulation and persistent bleeding. Under normal conditions, the platelet count in the human body ranges from 100 to 300 × 10⁹/L^[3]. However, in patients with ITP, the platelet count is far below 100×10⁹/L^[4]. Once the patient's platelet count falls below 50×10⁹/L^[3], the risk of bleeding will significantly increase.

Moreover, Professor Yang Renchi emphasized, “Bleeding caused by ITP is often insidious, spontaneous, and sudden. In daily life, patients frequently experience spontaneous bleeding or subcutaneous purpura; in severe cases, sudden major internal or intracranial hemorrhage may occur, posing a life-threatening risk.” He also reminded the public to pay greater attention to, understand, and take ITP seriously. As a benign condition, ITP is curable, and early detection and treatment are crucial. With active adherence to standardized treatment and efforts to minimize bleeding symptoms, patients can still enjoy a normal life.

Targeted Therapy: Supplement"Shortcomings" of Traditional ITP Treatment

Currently, in clinical practice, physicians predominantly employ first-line therapies, such as glucocorticoids and high-dose intravenous immunoglobulin (IVIG), to elevate patients’ platelet counts to safe levels and minimize the risk of bleeding events. Professor Yang Renchi points out that although these treatments can significantly increase platelet counts during initial therapy for most patients, long-term use is associated with an elevated risk of relapse, and the adverse effects may outweigh the benefits. For instance, glucocorticoids can cause adverse reactions in some patients, including osteoporosis, avascular necrosis of the femoral head, hypertension, and diabetes mellitus; excessive use in adolescent patients may even impair growth and development. Consequently, numerous therapeutic needs in clinical practice and among patients remain unmet.

“In recent years, the advent of targeted therapies has brought new hope to the treatment of immune thrombocytopenia (ITP). In particular, the introduction of eltrombopag, a thrombopoietin (TPO) receptor agonist that targets the pathogenesis of ITP, has addressed the limitations of conventional treatments. While meeting clinical and patient therapeutic needs, its oral convenience has further improved patient adherence to long-term therapy, ushering in a new era of sustained treatment for patients,” stated Professor Yang Renchi.。

Outpatient Chronic Disease Management: The “Key” to Long-Term Therapeutic Benefits for Patients

“The primary etiology of ITP is immune dysfunction, leading to reduced platelet production. Although medication can address the issue of platelet production in the short term, patients’ immune function cannot recover within a short period. Ultimately, the vast majority of ITP patients progress to chronic disease, requiring long-term maintenance therapy, which means the financial burden on patients will continue to increase,” stated Professor Yang Renchi.。

In recent years, the Chinese government has attached great importance to the prevention and treatment of hematologic diseases and malignant tumors, issuing corresponding policies to safeguard patients’ health. In 2019, immune thrombocytopenia (ITP), as one of the ten specified hematologic diseases and malignant tumors, was included in the first national list of pediatric diseases covered by medical treatment and security management programs. Furthermore, its corresponding oral innovative therapeutic agents were incorporated into the 2019 updated National Reimbursement Drug List.

“Historically, China lacked effective therapeutic agents for this condition; thanks to the national new drug review and approval system and the medical insurance framework, access to innovative treatments for this disease has been significantly improved.””Professor Yang Renchi emphasized, “In clinical practice, approximately 90% of patients with immune thrombocytopenia (ITP) can achieve satisfactory disease control through outpatient treatment. Outpatient medical insurance policies significantly influence clinical treatment decisions. The clinical pathway for ITP is clear and well-defined, aligning with the characteristics of diseases and treatments suitable for outpatient medical insurance management.” He called on various regions to incorporate ITP into the scope of outpatient reimbursement while implementing the 2019 updated National Reimbursement Drug List, taking into account local medical insurance conditions. This approach would yield significant clinical and economic benefits, conserve medical insurance funds, provide greater convenience for patients, facilitate long-term patient management, and help them return to normal life.。

*This material is intended to provide relevant knowledge in the disease area and enhance disease awareness, and is not for advertising purposes. The information contained herein is for reference only; please follow the advice or guidance of physicians or other healthcare professionals.

[1] Hemostasis and Thrombosis Group, Hematology Branch, Chinese Medical Association. Chinese Journal of Hematology. 2016;37(2):89-93.

[2] Hematology Group, Subspecialty Group of Pediatrics, Chinese Medical Association, et al. Chinese Journal of Pediatrics. 2013;51(5):382-384.

[3] Liang Hailing. Nursing Experience in the Process of Endotracheal Intubation for a Patient with Severe Thrombocytopenia and Respiratory Failure: A Case Report[J]. Guangdong Medical Journal, 2010, 31(4):481-481.

[4] Lou Yonggang, Wan Rugen. Methodological comparison of platelet parameter detection in patients with ITP[J]. Experimental and Laboratory Medicine, 2007, 25(4):401-401.