Struck By Tsunami: Difficult Case of a Severe Refractory ITP

Introduction

Immune thrombocytopenic purpura (ITP) is an autoimmune disorder characterized by a decreased platelet count and an increased risk of bleeding. While the majority of cases present with mild to moderate thrombocytopenia, severe cases can pose significant challenges in diagnosis and management.

We present the case of a 65-year-old male with severe ITP refractory to initial standard treatment including steroids and intravenous immunoglobulin (IVIG) requiring further management including rituximab, romiplostim, Fostamatinib, romiplostim, Efgartigimoid alfa-fcab , eltrombopag and partial splenic embolization.

Case presentation

Our patient is a 65-year-old male with a significant medical history, including end-stage renal disease (ESRD), diffuse large B-cell lymphoma (DLBCL) status post-stem cell transplant in 2015, and chronic immune thrombocytopenic purpura (ITP). He presented to the cancer center for his third rituximab infusion for ITP, during which he exhibited bloody vomitus. Blood work showed a hemoglobin was 5.8 g/dL, and a platelet count of 3 x 10^9 /L, while the remainder of his complete blood count (CBC) was within normal limits. Consequently, he was admitted briefly to the Medical Intensive Care Unit for gastrointestinal bleeding monitoring.

A computed tomography angiography (CTA) of the abdomen revealed no evidence of active extravasation. The patient received a total of 2 units of packed red blood cells (RBCs), and GI endoscopy was postponed due to his platelet count being less than 20 x 10^9 /L. The diagnosis of ITP was established in 2013, during hospital stay subsequent treatments included Decamethasone, intravenous immunoglobulin (IVIG), and rituximab at varying doses, none of which elicited a response. Additionally, the patient received Fostamatinib and Romiplostim, resulting in mild improvement in platelet count noted on day 6 of admission (platelet count: 17 x 10^9 /L).

On day 12 of admission, the patient underwent partial splenic artery embolization, resulting in a transient mild elevation of platelets to 13 x 10^9 /L, followed by a decline to 5 x 10^9 /L on day 17. A bone marrow biopsy conducted on day 19 revealed normocellular bone marrow with normal megakaryocytes and increased iron stores, along with rare ring sideroblasts. On day 33, the patient was initiated on Efgartigimoid alfa-fcab, but no improvement in platelet count was observed. A repeat splenic artery embolization was performed on day 56, followed by the commencement of eltrombopag on day 57. Subsequent to a splenic nuclear scan on day 65, residual active splenic tissue was identified. Von Willebrand factor (vWF) and factor 8 activity were both positive. Also during hospital stay General Surgery were consulted to assess the patient for splenectomy, which was deferred due to low platelet levels and to allow for further evaluation of the effects of embolization.

Following two weeks from the second partial splenic embolization, the patient's platelet count exhibited continuous improvement, reaching a level of 25 x 10^9 /L upon discharge.

Discussion:

Newer therapies are in the drug class thrombopoietin receptor agonists (TPO-RAs), which include romiplostim (Nplate), eltrombopag (Promacta), and avatrombopag (Doptelet), which have all been approved by the FDA. These medications are indicated for refractory ITP in patients who fail the initial therapies mentioned previously. TPO-RAs have a lower risk of side effects than older treatments, thus making them more tolerable for patients.

Although newer drug therapies have shown efficacy for many patients with early stages of ITP, our case demonstrates decreasing splenic function remains an effective treatment as it removes the major site of platelet phagocytosis and autoantibody production.15 However, due to critically low platelets in the majority of these patients, splenectomy can be associated with many complications including bleeding, infection, and thrombosis. Splenic artery embolization represents an effective alternative to increase platelet counts while minimizing complications.

Conclusion:

• This case underscores the challenge of managing severe Refractory Immune Thrombocytopenic Purpura in complex clinical contexts, necessitating further exploration of alternative therapeutic avenues.

• Platelets responsive to partial splenic artery embolization.

No relevant conflicts of interest to declare.