Observation, for skin bleeding, or immunomodulatory treatment, for severe bleeding, has been the standard care for children newly diagnosed with ITP. The majority of diagnosed children recover within 3-12 months after diagnosis, however, 20-25% develop chronic ITP. Previous studies proposed IV immunoglobin (IVIG) treatment as a therapy to decrease the risk of chronic ITP in children. Results obtained from limited randomized clinical trials have shown that IV immunoglobin treatment does not lead to lower rates of chronic ITP development in children. In these recent clinical trials, IV immunoglobin treatment displayed significant positive results early in treatment, in both complete and overall response, but after extended time resulted in similar outcomes to patients that did not receive treatment. Medical intervention can also lead to the risk of severe adverse events, such as epistaxis(bleeding from the nose) and menorrhagia(abnormally heavy bleeding at menstruation).
Katja M.J. Heitink-Polle, MD, from the department of pediatric hematology at University Medical Center Utrecht in The Netherlands, states that these trials show that observational intervention is a good alternative treatment and that IV immunoglobin treatment should be limited to accident prone toddlers or teenage girls who have had their menarche to stop or prevent bleeding. Additional research is needed to both predict patient response and recovery under IV immunoglobin treatment and also to develop more specific treatment management to better counsel patients and parents on the best possible treatment plan.
Heitink-Pollé KMJ, et al. Blood. 2018;doi:10.1182/blood-2018-02-830844. https://www.healio.com/hematology-oncology/hematology/news/in-the-journals/%7B40ea9df8-70cc-424a-aeb2-75eac9a5652f%7D/iv-immunoglobulin-does-not-prevent-chronic-immune-thrombocytopenia-among-children
Lusutrombopag (Mulpleta, Shionogi Inc), a thrombopoietin receptor agonist has been FDA-approved for thrombocytopenia in adults with chronic liver disease before undergoing a medical or dental procedure. This therapy is delivered as a seven day dosing regimen 8-14 days prior to surgery and within 2-8 days after completion of the last dose to prevent and control bleeding. Lusutrombopag will greatly reduce the need for platelet transfusions before and after surgical procedures in patients with chronic liver disease.
This therapy was analyzed and approved through phase 3 clinical trials (studies efficacy in hundreds of patients in a randomized, double-blind, and placebo-controlled trials), where the most common adverse reaction was a headache. However, this drug treatment has not been approved for normal administration in hopes of normalizing platelet counts. Future studies, may investigate the use of Lusutrombopag in patients suffering from conditions with lower platelet counts, such as ITP, but current studies do not approve using Lusutrombopag to normalize platelet count.
Brooks, Megan. FDA OKs Lusutrombopag for Thrombocytopenia in Adults With Liver Disease - Medscape - Jul 31, 2018. https://www.medscape.com/viewarticle/900052?nlid=124151_3901&src=wnl_newsalrt_180731_MSCPEDIT&uac=125458BZ&impID=1700172&faf=1