Rituximab versus splenectomy in persistent or chronic adult primary immune thrombocytopenia: an adjusted comparison of mortality and morbidity

Abstract

Splenectomy and rituximab are both recommended as second-line treatment in immune thrombocytopenia (ITP), but they have never been directly compared. We compared their efficacy and serious adverse outcomes in a retrospective cohort of 105 adult primary ITP patients exposed to one or other of these treatments. Primary outcome was composite: death from hemorrhage or from infection and hospitalization for bleeding or for infection. Secondary outcomes were overall mortality, hospitalization for bleeding, hospitalization for infection, as well as response and complete response (international definitions). Analyses were adjusted on a propensity score. Patients treated with rituximab (n 5 43) were older and had more comorbidities than the splenectomized patients (n 5 62). Mean follow-up was, respectively, 3 and 8.4 years. After adjustment on the propensity score, there was no difference between the two groups regarding the primary and other clinical outcomes. In the multivariate analysis, only a history of mucosal bleeding (HR 3.2 95% CI [1.2-8.5]) and a Charlson score 1 (HR 4.2 95% CI [1.8-9.6]) were associated with the primary outcome. These two factors were also associated with hospitalization for bleeding. As expected, response, complete response and maintenance rates were higher in the splenectomy group. Splenectomy compared with rituximab was independently associated with a response at 12 months (OR 4.4, 95% CI [1.7-11.8]). Then, adjusted analyses in this real-life cohort confirmed the better results of splenectomy compared with rituximab.

Long-term outcome following splenectomy for chronic and persistent immune thrombocytopenia (ITP) in adults and children

“…This long remission rates have been described in other series with similar long-term remission rates ranging from 60-80 % [21][22][23][24]. Thus, even in the presence of excellent remission rates with the use of anti-CD20 monoclonal antibodies such as rituximab, it is still debatable whether the use of such therapies are superior to splenectomy since monoclonal antibodies are also associated with risks such as infections [25,26]. The cost associated with the use of rituximab inhibits the widespread use of such drugs in developing countries, and hence, splenectomy is still a cheap but very effective option for patients with relapsed/refractory ITP.…”

Immune thrombocytopenia: Effectiveness of frontline steroids and comparison of azathioprine, splenectomy, and rituximab as second‐line treatment

“…Splenectomy has been compared with rituximab in a retrospective data analysis. 13 The study revealed superior survival and durable remission in favor of splenectomy. However, due to its retrospective, non-randomized nature, the demographic data and comorbidity status between patients undergoing splenectomy and patients treated with rituximab were not balanced.…”

“…In modern clinical trials, TPO‐RAs have been compared with placebos or investigator defined “standard of care”, rather than specific second‐line treatment agents. Splenectomy has been compared with rituximab in a retrospective data analysis . The study revealed superior survival and durable remission in favor of splenectomy.…”

“…and TPO‐RAs, are available as treatment options. Clinical data comparing the outcome of different treatment modalities are limited . Therefore, it is often difficult for patients and physicians to make decisions based on limited evidence.…”

“…Clinical data comparing the outcome of different treatment modalities are limited. 13 Therefore, it is often difficult for patients and physicians to make decisions based on limited evidence. We aimed to investigate the outcome of frontline steroid treatment for ITP patients and compare the common second line treatment modalities in a large cohort in Taiwan.…”