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Summary:To determine the role of allogeneic, autologous and syngeneic hemopoietic stem cell transplantation (SCTx) as a treatment for severe autoimmune disease (AID) we performed a literature search employing Medline, Cancer Lit and abstract books for reports on transplants for blood disorders and a concomitant AID. All reviews, case reports and abstracts available between June 1977 and September 2001 were used and attempts made to update them by e-mail by the corresponding authors. Disease-free survival (DFS) after allogeneic SCTx for 23 patients with severe aplastic anemia was 78% at 16 years and survival in unmaintained remission of concomitant AID was 64% at 13 years. DFS after allogeneic SCTx for 24 patients with hematologic malignancies was 87% at 15 years and survival in unmaintained remission for concomitant AID was 70% at 11 years. DFS after autologous SCTx for 24 patients with hematologic malignancies was 48% at 6 years and survival in unmaintained remission for concomitant AID was 29% at 3 years. Among 30 patients given allogeneic SCTx 19 developed graft-versus-host disease (GVHD) and 11 did not. Upon clinically justified discontinuation of all immunosuppressive therapy, 3/11 patients without GVHD relapsed with their concomitant AID (freedom of AID-relapse 69% at 9 years), whereas none of 19 patients with GVHD did so (log rank: P ‫؍‬ 0.0135). Freedom of AID-relapse was superior after allo SCTx compared to autologous SCTx (89% at 18 years vs 38% at 5 years; log rank: P ‫؍‬ 0.0002). Our data suggest that a graft-versus-autoimmunity effect after allogeneic hemopoietic SCTx mediates elimination of autoimmunity.

Most patients with aplastic anemia recover bone marrow function after receiving a transplant from a genetically identical twin. Pretransplantation conditioning may increase the chance of bone marrow recovery but does not seem to improve survival.

Five patients with severe aplastic anaemia (SAA) who, simultaneously (n = 3) or consecutively (n = 2), presented with multiple sclerosis (MS) (n = 2) or immune hyperthyroidism (IHT) (n = 2) or subacute thyroiditis (n = 1) are described. Two female patients with MS developed SAA after a small dose of azathioprine. Another patient simultaneously presented with IHT and SAA. SAA and MS responded to cyclosporine while IHT required ¹³¹I. Relapsing SAA in 1 patient with MS was treated with antithymocyte globulin (ATG) which induced acute exacerbation of MS. Despite the low total dose of ATG (31.5 mg/kg), complete remission of SAA was obtained. Two other patients developed thyroid disorders, 42 and 106 months after successful immunosuppression with ATG/high-dose methylprednisolone. IHT and subacute thyroiditis were successfully treated with ¹³¹I or prednisolone, respectively, without recurrence of SAA in both cases. These are the first documented cases of SAA evolving in the course of MS while the coincidence with IHT was already described. Since enhanced expression of interferon-γ plays a crucial role in SAA as well as in MS and in IHT, similar pathogenetic principles may apply for these seemingly unrelated disorders.

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