On the Pathogenesis and Treatment of Aplastic Anemia

Bone Marrow Transplantation for Severe Aplastic Anemia

“…First, our re sults, both with bolus methylprednisolone [2] and with ALG, confirm Speck's conten tion [15] that SAA is not uniformly a defect of the earliest hemopoietic progenitor cell, and that, at least in some cases, it may be an autoimmune condition not so distant from pure red cell anemia (PRCA), depending on the cellular target (stem cell in SAA, an un identified erythroid precursor in PRCA) against which the immunologic reaction is mounted [7,8].…”

“…In our patients mar row reconstitutions were clearly autologous as proved by means of chromosome studies and red cell phenotypes. The reversal to normal blood and marrow parameters in 3 cases, as in a recent much larger series [15] would suggest that engraftment is not a prerequisite to ensure long-term survival as previously reported [11].…”

“…4 patients were condi tioned with ALG and received bone marrow from a haploidentical sibling or parent: 1 patient was refractory; 3 patients showed evidence of hematologic reconstitution, but 2 of these required a second course of ALG. 3 patients in this group are alive between 60 and 490 days; 1 patient died on day 121 of HBSAg-negative acute hepatitis.Bone marrow transplantation (BMT) has proven to be superior to conventional an drogen-corticosteroid therapy in the man agement of severe aplastic anemia (SAA) [20] and encouraging results have been re ported from several institutions [1,5,6,15,18,19]. Nevertheless, major problems such as graft rejection and graft-versus-host dis ease (GvHD) are still responsible for a 60% mortality rate [16] and need to be solved.…”

Inhibition of CFU_E and BFU_E by Mononuclear Peripheral Blood Cells during Chronic Benzene Treatment in Rabbits

“…A possible in vivo correlate of our find ing is the demonstration that in mice the BM microenvironment is affected by ben zene poisoning [2], while the stem cells are spared [2,13]. They may also offer an ex planation of earlier data by Speck et al [14], who found that benzene-treated aplas tic rabbits can be cured by administration of a combination of antilymphocyte serum and allogeneic BM.…”

Bone Marrow Transplantation

“…The results of marrow grafts between HLA-non-fully-identical siblings continue to be frustrating in SAA [59], In 1977 we reported the results of 29 patients with SAA without HLA-identical siblings treat ed with antilymphocyte globulin (ALG) with or without marrow infusion [60], The rationale and the experimental and clini cal bases for this therapeutic approach have been outlined [61][62][63]. In 1981 we published the results of 50 patients with SAA treated and evaluated in a prospec tive study according to one protocol com paring BMT with ALG treatment aiming at autologous hemopoietic reconstitution [64], This study showed that therapy with ALG is at least as good or better than BMT.…”