High‐dose dexamethasone plus recombinant human thrombopoietin vs high‐dose dexamethasone alone as frontline treatment for newly diagnosed adult primary immune thrombocytopenia: A prospective, multicenter, randomized trial

Abstract

contributed equally to this study. This study is registered as clinicaltrials.gov identifier: NCT01734044.

Recombinant Human Thrombopoietin Accelerates the Recovery of Platelet in Patients With Lower-Risk Myelodysplastic Syndrome: A Proof-of-Concept Study

“…As for disease progression, more platelet deterioration was noted in the non-rhTPO group, although not significant. In addition, bone fibrosis was not observed in our study, as verified by other studies showing that rhTPO did not increase transformation to MDS and the degree of fibrosis in patients with AA (21,25).…”

“…TPO is an endogenous factor that regulates the proliferation and maturation of megakaryocytes and the generation of platelets, which plays a biological role by binding to c-MPL, a specific receptor on the surface of hematopoietic stem cells and megakaryotic progenitor cells and activates the signaling pathway of Janus kinase 2 (JAK2)/signal transducer and activator of transcription 3 (STAT3) (21,(25)(26)(27). In this proofof-concept study, patients newly diagnosed with LR-MDS were enrolled.…”

“…We assumed that the platelet response in patients treated with rhTPO might be 1.3 times that of the controls (based on the experience of rhTPO in newly diagnosed adult primary ITP) (21). Power calculation indicated that if we want to have an 85% chance of rejecting the hypothesis with no difference at the level of 0.05, we needed 62 patients in each group.…”

“…Recombinant human thrombopoietin (rhTPO), a glycosylated full-length peptide TPO produced by 3SBIO (Shenyang, China), has shown a high response rate (60.3%-66.7%) in corticosteroidresistant or relapsed ITP patients (18)(19)(20)(21). Although patients developed transient anti-TPO antibodies after rhTPO treatment, the antibodies did not have the activity of neutralizing endogenous TPO (22).…”

Early combination treatment of immune thrombocytopenia: Is this the way?

“…There are three reasons why the study “High‐dose dexamethasone plus recombinant human thrombopoietin vs high‐dose dexamethasone alone as frontline treatment for newly diagnosed adult primary immune thrombocytopenia: a prospective, multicenter, randomized trial” by Yafei Yu et al, 1 combining dexamethasone and recombinant human thrombopoietin and comparing the combination to dexamethasone alone, may come to be considered a landmark paper on the treatment of adults with immune thrombocytopenia (ITP): It describes with more careful attention to detail than virtually any other study of this type the clinical characteristics and key laboratory findings in the patients and their outcomes. This includes not only short, middle and long‐term outcomes but also why some patients are lost to follow up, drop out due to need for other therapy, or drop out by choice. The study suggests that treating ITP more aggressively at diagnosis with the agents chosen (dexamethasone + rhuTPO 2‐4 ) may improve the rate of long‐term remission with minimal additional side effects combined with good short term response. It illustrates how academic hematologists in China can utilize their large number of ITP patients to conduct adequately‐powered, randomized studies difficult to perform elsewhere. …”