Effectiveness of Second-Line Treatment in Patients with Steroid-Refractory Immune Thrombocytopenic Purpura

Amima Sabir; Jahanzeb Ur Rehman; Mahnoor; Afsheen Liaqat; Mehreen Ali Khan; Raheel Iftikhar

Authors

Amima Sabir Department of Clinical Hematology and Bone Marrow Transplant. National University of Medical Sciences, Rawalpindi, Pakistan
Jahanzeb Ur Rehman Department of Clinical Hematology and Bone Marrow Transplant. National University of Medical Sciences, Rawalpindi, Pakistan
Mahnoor Department of Clinical Hematology and Bone Marrow Transplant. National University of Medical Sciences, Rawalpindi, Pakistan
Afsheen Liaqat Department of Clinical Hematology and Bone Marrow Transplant. National University of Medical Sciences, Rawalpindi, Pakistan
Mehreen Ali Khan Department of Clinical Hematology and Bone Marrow Transplant. National University of Medical Sciences, Rawalpindi, Pakistan
Raheel Iftikhar Department of Clinical Hematology and Bone Marrow Transplant. National University of Medical Sciences, Rawalpindi, Pakistan

Keywords:

Immune thrombocytopenia (ITP), Thrombopoietin receptor (TPO), overwhelming post splenectomy infection (OPSI).

Abstract

Objective: To analyze and compare the data of ITP patients who received Eltrombopag and who underwent splenectomy and present outcome in these patients.

Methodology: Study was conducted retrospectively at tertiary care hospital in Rawalpindi, between 2019 and 2023. Inclusion criteria included patients who were steroid resistant, refractory or steroid dependent, and received Eltrombopag or underwent Splenectomy. WHO Bleeding Scale was used to asses bleeding score and response assessment was performed according to the American Society of Hematology’s 2009 International Working Group Report.

Results: A total of 40 patients were included in the study, with a mean age of 24.8 years (Range 2-64) with male-to-female ratio of 1.5:1. The selected patients were divided into two groups: N=20 in the splenectomy group (Cohort 1) and N=20 in the Eltrombopag group (Cohort 2). All patients received oral steroids at 1 mg/kg dose as first line treatment. Both groups showed an overall response rate of 29 (72.5%), while 11(27.5%) had no response. Of those who responded, 17 (58.6%) belonged to Cohort 1 and 12 (41.4%) to Cohort 2. In Cohort 1, 15 patients (88.2%) achieved a complete response, and 2 (11.8%) had a partial response. In Cohort 2, complete response was seen in 10 patients (83.3%) and partial response in 2 (16.7%). There was a significant association of age with response. Major complications and OPSI was not seen in any patient. Among 40 patients only 4 (10%) relapsed post second line treatment.

Conclusion: Both splenectomy and Eltrombopag are effective as second line treatment options for steroids refractory ITP with fewer relapses and minimal side effects, however higher proportion of patients achieved complete response in the Splenectomy group compared to Eltrombopag group