Long-Term Results of the Immunosuppressive Treatment of Patients with Severe Acquired Aplastic Anemia: A Single Institution Study

Abstract

In México, only few patients can afford adequate treatment for aplastic anemia. In a single institution 61 individuals with severe aplastic anemia (SAA) were identified; of these, 33 were followed for at least 3 months, 26 could be treated with immunosuppression (IS), 20 with antithymocyte globulin and cyclosporin A (ATG + CyA) and 6 with CyA, whereas 7 individuals were given only anabolic androgens. In the patients treated with IS a complete remission was achieved in 12 and a partial remission in 6, the overall response rate being 69%. The 5,729-day overall survival (SV) was 54%; it was superior for the patients who received both ATG and CyA (58 vs. 50%). These data contrast with those of the patients who were given only androgens: 14% SV at 5,510 days. Within the group of patients who received IS, one developed acute myelogenous leukemia, another paroxysmal nocturnal hemoglobinuria and another a primary Sjögren’s syndrome-associated low-grade, B cell, non-Hodgkin’s lymphoma. Two individuals who received IS were allografted from HLA-identical siblings and survived 70 and 413 days after the allograft. On the other hand, we also found that, for several reasons, mainly economic ones, only 41% of individuals with SAA finally benefited from the best immunosuppressive therapeutic approach. IS was a good therapeutic choice for some patients with SAA, clearly better than androgen treatment.

SINGLE-CENTER EXPERIENCE: Immunosuppressive Therapy as Frontline Treatment for 33 Children with Acquired Severe Aplastic Anemia

“…Barring specific contraindications, patients with SAA should proceed straight to HSCT if they have HLA-identical sibling donors [18]. However, Our study demonstrated overall survival equivalent to that previously reported for HLA-identical BMT recipients [19,20], which is superior to that reported for those treated with IST alone [21,22]. An increased risk of evolution to myelodysplastic syndrome/acute myeloblastic leukemia has been observed in long-term survivors of aplastic anemia not treated with HSCT [23].…”

Allografting in patients with severe, refractory aplastic anemia using peripheral blood stem cells and a fludarabine-based conditioning regimen: The Mexican experience

“…Patients met the criteria for the diagnosis of severe AA [1,6]. All patients had failed to respond to conventional treatment with immunosuppression using ATG and/or cyclosporin and had been heavily transfused (see Table I).…”

“…Mexico is a country with a large number of cases of AA [6]; however, due to financial and idiosyncratic factors, the elapsed time from diagnosis to hematopoietic cell transplantation is usually long, leading to AA patients being multiply transfused and highly sensitized to HLA antigens before transplant is undertaken. We have studied the effectiveness of peripheral blood stem cells and a fludarabine/cyclophosphamide-based conditioning regimen without ATG or lymph node irradiation in 23 AA patients who had no response to previous conventional pharmacologic treatment, including androgens and immunosuppressive therapy, and who had been heavily transfused.…”

Lymphoproliferative Disorders after Immunosuppressive Therapy for Aplastic Anemia: A Case Report and Literature Review

“…For determining the incidence of LPD after IST, we searched Medline (Pubmed) for published articles about the long-term results of IST for AA [1, 4, 5,7,8,9,10,11,12,13,14,15,16,17,18]. We found 3 cases with LPD among the published studies [5, 7, 12].…”