Cyclosporine monotherapy for severe aplastic anemia: a developing country experience

Abstract

BACKGROUNDImmunosuppression is the most effective treatment for aplastic anemia after hematopoietic stem cell transplantation. Although the combination of cyclosporine and antithymocyte globulin (ATG) is superior to either agent alone, cyclosporine monotherapy is an easily available, safe and cheap immunosuppressive therapy (IST) option. These advantages are particularly valuable in developing countries where ATG is frequently not available.PATIENTS AND METHODSIn the referral hematology center in Yemen, 20 patients (16 males and 4 females) with severe aplastic anemia (SAA) were prospectively identified and managed with cyclosporine monotherapy during the period between April 2001 and November 2004.RESULTSData from 14 patients who received cyclosporine for at least 3 months were analyzed. At 6 months, 2 (14.3%) patients achieved complete remission (CR) and 5 (35.7%) patients achieved partial remission (PR) and at 1 year, 4 (28.6%) patients achieved CR and 3 (21.4%) patients remained in PR. The overall response rate was 50% and the cumulative survival rate at 1 year was 78.6%. The median time to remission was 120 days (range, 46 to 131 days). Side effects were modest and easily monitored.CONCLUSIONOur results support findings that cyclosporine monotherapy is an effective and safe immunosuppressive therapy for SAA, and that it could be a reasonable IST option for patients in developing countries.

Special issues related to the diagnosis and management of acquired aplastic anemia in countries with restricted resources, a report on behalf of the Eastern Mediterranean blood and marrow transplantation (EMBMT) group and severe aplastic anemia working party of the European Society for blood and marrow transplantation (SAAWP of EBMT)

“…Cyclosporine is typically used in combination with ATG as IST, but due to nonavailability of ATG particularly horse ATG, financial constraints and lack of adequate blood components and other support facilities; it has been used as a single agent for treatment of acquired AA [63]. Most of the available evidence of CsA monotherapy outcomes come from small single center reports.…”

Pattern of haematological diseases diagnosed by bone marrow examination in Yemen: a developing country experience

“…The epidemiological pattern in those regions probably do not apply to our country and possibly to other middle east countries although data from these countries have not been reported. The immunopathogenesis of our aplastic anaemic patients has been confirmed by their response to cyclosporin monotherapy (Al‐Ghazaly et al. , 2005).…”

“…Aplastic anaemia was the most common and was diagnosed in accordance with the well‐known criteria (Beutler et al. , 1995; Al‐Ghazaly et al. , 2005).…”

Alternative treatment modality for severe aplastic anemia in a resource-limited setting: a single-institution prospective cohort study from Upper Egypt

“…Our results differ from those found in studies of CsA treatment for adults with SAA. Shetty et al recorded a 56.2% response rate to CsA in 20 adult patients with SAA after 3 months39, while Ghazaly et al found a 50% response in adults with SAA at 6 months [ 15 ].…”

“…CsA is sometimes used alone as a monotherapy in countries with poor resources [ 15 , 16 ]. It is an effective immunosuppressant, inexpensive, accessible, can be administered to outpatients and is less toxic than combined treatment with ATG.…”