Aplastic Anemia: Pathogenesis and Treatment

Abstract

Treatment of severe aplastic anemia (SAA) in Europe between 1970 and 1986 is reviewed. 487 patients received an HLA-identical BMT: results are encouraging and currently suggest a 65% survival. However, many patients cannot be offered this procedure because of the absence of an appropriate donor. Forty-five patients were given a non-HLA identical BMT: results are dependent on the degree of mismatch. Immunosuppression (IS) was given to 509 patients: 50% of these survive. Some mechanisms regulating in vitro hematopoiesis are discussed, together with their relevance in the treatment of SAA.

Alternative immunosuppression in patients failing immunosuppression with ATG who are not transplant candidates: Campath (Alemtuzumab)

“…7,8 Current standard IST for AA patients consists of ATG þ CYA, with an expected 60-70% probability of response and 70-80% OS at 1 year. 6,9,10 Both horse ATG preparations (ATGAM (Upjohn, Kalamazoo, MI, USA), 40 mg/kg per day for 4 days 11,12 or Lymphoglobuline (Sanofi, Paris, France), 15 mg/kg per day for 5 days [13][14][15] ) seem equally effective for the treatment of AA; in contrast, rabbit ATG (Thymoglobulin (Sanofi), 3.5 mg/kg per day for 5 days) has been shown inferior to horse ATG in a large randomized study, 16 as well in a pilot study from the Severe Aplastic Anemia Working Party (SAAWP). 17 Thus, horse ATG þ CYA remains the preferred first-line IST for AA patients not eligible for a SCT from a sibling HLA-matched donor.…”

Polimorfismo genético da glutationa S-transferase mu1 e theta 1 em pacientes com anemia aplástica adquirida: uma experiência brasileira

“…No entanto, após vários anos e a evidên-cia clínica bem consolidada da resposta da maioria destes pacientes à imunossupressão, o entendimento da fisiopatologia desta doença levou a uma hipótese de um distúrbio auto-imune. 3 Além disto, nas séries mais modernas, poucos pacientes apresentam história de exposição a estes agentes. No entanto, há ainda o dilema relacionado àqueles que não respondem à imunossupressão.…”