Abstract

Objectives: Rituximab (RB) is an anti-CD20 murine/human chimeric antibody that has been used in the treatment of auto-immune diseases, including immune thrombocytopenic purpura (ITP). The effectiveness of RB in the treatment of patients with ITP has been evaluated.

Patients and methods: A multicentric retrospective study of the patients with ITP treated with RB has been carried out. A questionnaire about clinical characteristics and response to RB was sent to the participating centers. These questionnaires were filled out by the physicians in charge of each patient. We received questionnaires from 43 centers The response criteria were those published by

Stasi R et al (
Blood
2001
;
98
:
952
)
: Complete Response (CR): platelets >100 x 109/l; Partial Response (PR): 50–100 x 109/l; Minimum Response (MR): 30–50 x 109/l. Data from 99 patients were analyzed. Ninety two out of 99 patients were evaluated. The median age at diagnosis was 54 years (4–98), 56% were women. 55.4% of the cases were idiopathic ITP, 14.1% were associated with B-CLL, 8.7% with auto-immune diseases and 3.3% with lymphoma. Before RB they have received several treatment schemes including steroids (98%), IVIG (88%) and cyclophosphamide (26%). 52.2% were splenectomized. Median of platelets before treatment with RB was 8 x 109/l (1–30), 66.3% had less than 10 x 109/l. 87% of the patients were treated with 4 doses of RB (1–6), while 34 received it in association with other treatments, mainly steroids.

Results: 42 patients obtained CR (46.2%) and 8 patients obtained PR (8.8%), a total of 50 global responses (55%). The majority of the responses took place in the first week (26% out of the total, 40% of the responders). The maximum response was also soon achieved, median of 5 weeks, with platelets of 141 x 109/l (10–651). There were no differences in the response between splenectomized or not splenectomized patients. 29 patients maintained the CR (69% of responders), with a monitoring median of 9 months (2–42). The toxicity of the treatment was minimum: 2 fever episodes with the infusion and 2 skin eruptions.

Conclusions: This is the widest series of patients with ITP treated with RB. These results suggest that RB can be considered as a rescue treatment for patients with refractory ITP to steroids and/or splenectomy, with an excellent tolerance. Further monitoring is needed in order to establish the real role of RB as a rescue treatment for refractory ITP.

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