Ivy P. Altomare, MD, associate professor of medicine, Department of Medicine, Duke University School of Medicine, and medical oncologist, Duke Cancer Network, discusses investigational agents in immune thrombocytopenia (ITP).

There are many emerging classes of agents in the ITP pipeline including neonatal Fc receptor (FcRn) inhibitors, BTK inhibitors, and low-dose decitabine, says Altomare. The FcRn inhibitors are administered intravenously or subcutaneously.

Phase 2 data presented at the 2019 ASH Annual Meeting demonstrated efficacy with the FcRn inhibitors efgartigimod (ARGX-113) and rozanolixizumab (UCB7665) in ITP, explains Altomare. As such, phase 3 clinical trials with these agents are ongoing.

FcRn inhibitors can target the FcRn pathway, which is a key part of preserving and recycling immunoglobulin, says Altomare. Inhibition of the FcRn pathway can degrade an IgG molecule, thus making these drugs potentially effective in treating patients with autoimmune diseases, such as ITP.

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Ivy P. Altomare, MD, discusses investigational agents in immune thrombocytopenia.

Dr. Altomare on Investigational Agents in ITP

Ivy P. Altomare, MD, associate professor of medicine, Department of Medicine, Duke University School of Medicine, and medical oncologist, Duke Cancer Network, discusses some of the challenges of treating immune thrombocytopenia (ITP).

ITP requires a clinical diagnosis because there is no definitive test that can be used to diagnosis ITP, says Altomare. In addition to diagnosis, treatment for highly refractory patients with ITP can be challenging, explains Altomare. As the most effective agents are used upfront, there are limited options for patients who relapse.

Patients who have active bleeding or a strong risk of bleeding in addition to a blood clot are a particularly challenging group of patients to treat, says Altomare. In these cases, it is important to establish a balance between preventing thrombosis and preventing the risk of bleeding.

Thrombopoietin receptor agonists that are frequently utilized in the treatment of ITP predispose patients to deep vein thrombosis and venous thromboembolism (VTE), says Altomare.

However, fostamatinib disodium hexahydrate (fostamatinib; Tavalisse) does not appear to increase the risk of thrombosis and should be considered for patients who are at risk of developing VTE, concludes Altomare.

Ivy P. Altomare, MD, discusses some of the challenges of treating patients with immune thrombocytopenia. 


Dr. Altomare on the Challenges of Treating ITP

Ivy P. Altomare, MD, associate professor of medicine, Department of Medicine, Duke University School of Medicine, and medical oncologist, Duke Cancer Network, discusses treatment options for patients with immune thrombocytopenia purpura (ITP).

Steroids are a standard of care for patients with ITP in the first-line setting, says Altomare. Steroids are effective in most patients. However, only 30% of patients will maintain their response at 2 years. As such, most patients will relapse and require second-line therapy.

Several thrombopoietin receptor agonists are approved for the treatment of patients with chronic ITP, including eltrombopag (Promacta), romiplostim (Nplate), and avatrombopag (Doptelet), says Altomare. Importantly, these agents do not cause long-term immunosuppression.

Additionally, fostamatinib disodium hexahydrate (fostamatinib; Tavalisse) is a well-tolerated, oral spleen TKI that has demonstrated significant activity in patients with ITP, says Altomare.

These agents have not been compared head-to-head. Additionally, there are no predictive biomarkers to guide treatment selection and sequencing. As such, treatment selection may be influenced by patient preference, physician experience, and payers, concludes Altomare.

Ivy P. Altomare, MD, discusses treatment options for patients with immune thrombocytopenia purpura.



Dr. Altomare on Treatment Options in ITP

Ivy P. Altomare, MD, associate professor of medicine, Department of Medicine, Duke University School of Medicine, and medical oncologist, Duke Cancer Network, discusses the management of patients with immune thrombocytopenia purpura (ITP). 

Typically, ITP is a chronic disease that affects adult patients, says Altomare. Although relapse is common, patients can live with the disease if their platelet counts are maintained above a certain level.   

Notably, patients with ITP are at an increased risk of serious bleeding, says Altomare. Additionally, ITP can lead to spontaneous intracranial hemorrhage in rare instances. 

Currently, multiple novel therapies are available to treat patients with ITP. These treatments may mitigate those risks and reduce the long-term immunosuppression that was associated with historical treatments, concludes Altomare.

Ivy P. Altomare, MD, discusses the management of patients with immune thrombocytopenia purpura.



Ivy P. Altomare, MD

Articles

Dr. Altomare on Investigational Agents in ITP

Dr. Altomare on the Challenges of Treating ITP

Dr. Altomare on Treatment Options in ITP

Dr. Altomare on the Management of ITP