Re‐evaluation of the role of azathioprine in the treatment of adult chronic idiopathic thrombocytopenic purpura: a report on 53 cases

Abstract

We treated 53 adults (mean age 54 years, range 17-89; 37 females and 16 males) with chronic idiopathic thrombocytopenic purpura (ITP) by azathioprine. All patients had received at least one form of therapy (including splenectomy in 40 patients) and had less than 50 x 10(9)/1 platelets. The duration of ITP before azathioprine was started ranged from 6 to 350 months (median 19). All patients initially received 150 mg/d of azathioprine. This was associated with a short initial course of prednisone (0.3-0.5 mg/kg d) in 10 of them, who were refractory to prednisone alone. 34 (64%) patients responded, including 24 (45%) complete remissions (CR), three (6%) partial remissions (PR) and seven (13%) minor responses (MR). Median time to achieve response was 4 months. 17 of the CR persisted after 7-182 months, 10 of them after discontinuation of azathioprine. Seven patients relapsed after 4-26 months, five of them after azathioprine was stopped or its dose was reduced. PR were short and the median duration of MR was 8 months. Overall, 21 patients (40%) had responses lasting 1 year or more and 17 (32%) lasting 2 years or more. Median duration of treatment was 18 months (range 3-84). Five patients died of bleeding while on treatment. No prognostic factors for response to azathioprine were found. Mild leucopenia was seen in seven patients and a moderate (x3) increase in transaminases in two patients. No opportunistic infections were seen and no malignancy has occurred since the onset of azathioprine. We conclude that azathioprine gives a relatively high incidence of durable responses and very limited side effects in chronic ITP, when splenectomy has failed or is contraindicated. This efficacy, in our experience, is superior to that obtained with other therapeutic approaches. As responses may be delayed, a course of azathioprine of 4 months is required before one can infer a failure to respond. In responding patients, however, the optimum duration of treatment remains to be established.

Dapsone salvage therapy for adult patients with immune thrombocytopenia relapsed or refractory to steroid and rituximab

“…Furthermore, some patients have contraindications or refuse surgical intervention, and there is still some concern regarding the possible long-term increased risk of infectious complications. Several other immune suppressive agents as azathioprine [10,11], cyclosporin-A [12], micophenolate mofetil [13,14], some cytotoxic drugs as cyclophosphamide [15,16] or vincristine [17] and some hormonal agent as danazol [18,19] may be active in 30-50% of patients with ITP, but their administration is frequently associated with side effects. If compared with these multiple agents, dapsone appears characterized by similar, if not higher, therapeutic activity and a much more favorable safety profile.…”

Maintenance Therapy with Rituximab in Adult Patients with Immune Thrombocytopenia

“…About 40% of splenectomized patients either do not respond or relapse after surgery [6], approximately 10% of patients have early perioperative or delayed infections, and some develop surgical complications, although rarely fatal. In case of relapse or resistant to corticosteroids ITP, other immunosuppressive drugs can be used including azathioprine, cyclosporine A, cyclophosphamide, danazol, mycophenolate mofetil, all with marginal efficacy [7][8][9][10][11]. Intravenous immunoglobulin (IVIG) and anti-D are also used in the treatment of ITP, especially when rapid improvement of platelet count is needed (in case of life-threatening bleeding or prior to splenectomy or other surgical procedures).…”

Management of Idiopathic Thrombocytopenic Purpura in Adult: A Review Article

“…Another review 27 of the use of azathioprine 150 mg per day for a median of 18 months shows Platelet responses in 64% of patients and were complete in 45% of these.…”