Haematopoetic stem cell transplantation for refractory autoimmune cytopenia

Abstract

Summary This study describes the outcome of patients receiving haematopoietic stem cell transplantation (HSCT) to treat severe refractory autoimmune cytopenia. The registry of the European Group of Blood and Marrow Transplantation holds data on 36 patients receiving 38 transplants, the first transplant was autologous for 27 and allogeneic for nine patients. Patients had autoimmune haemolytic anaemia (autologous: 5; allogeneic: 2), Evans's syndrome (autologous: 2; allogeneic: 5); immune thrombocytopenia (autologous: 12), pure red cell aplasia (autologous: 4; allogeneic: 1), pure white cell aplasia (autologous: 1; allogeneic 1), or thrombotic thrombocytopenic purpura (autologous: 3). Patients had longstanding disease having failed multiple prior treatments. Among 26 evaluable patients mobilized for autologous HSCT, three died of treatment‐related causes, one died of disease progression, seven were non‐responders, six patients had transient responses and nine had continuous partial or complete remission. Of the seven evaluable patients receiving allogeneic HSCT, one died of treatment‐related complications, one with transient response died of progressive disease and five had a continuous response. Autologous and allogeneic HSCT may induce a response in a subset of patients with autoimmune cytopenia of long duration albeit at the price of considerable toxicity.

Successful treatment of pure red cell aplasia with autologous stem cell transplantation

“…However, no erythropoietic response followed the mobilization and the patient relapsed at day 104. Although accompanied by no specific details, a recent report from the European Group of Blood and Bone Marrow Transplantation lists four patients with PRCA who underwent autologous transplantation; two had no response and two had a transient response [6]. This appears to be the first detailed report of primary acquired PRCA that was successfully treated by autologous HSCT and achieved a continuous response.…”

“…For these patients, the treatment strategies remain ill-defined. Allogeneic HSCT has been used with some success in those patients with relapsing PRCA or PRCA refractory to the conventional modalities [3][4][5][6]. Certainly, considering their mechanism, allogeneic transplants are more promising in achieving a complete remission, because they may also exert a GVA effect by gradually eradicating the recipient's lymphopoiesis [7].…”

“…Because autologous transplants offer a greater safety profile, they have been utilized more extensively for autoimmune diseases such as systemic lupus erythematosus (SLE), Crohn's disease, multiple sclerosis, [7,8] and hematologic diseases such as severe immune thrombocytopenic purpura [6,9]. Autologous HSCT is thought to act through two mechanisms: powerful immunosuppression and possible ''resetting'' of the immune system and [7,10] by allowing lymphoid reconstitution that is tolerant to the autoantigens that had previously caused the autoimmune process [11].…”

“…Autologous HSCT is thought to act through two mechanisms: powerful immunosuppression and possible ''resetting'' of the immune system and [7,10] by allowing lymphoid reconstitution that is tolerant to the autoantigens that had previously caused the autoimmune process [11]. There have been four reports of successful allogeneic transplants for treatment of PRCA [3][4][5][6] but none with an autologous transplant. Musso et al attempted an autologous HSCT in a 29-year-old patient with chronic acquired PRCA refractory to conventional immunosuppressive therapies after mobilization with Cy 4 g/m 2 and G-CSF 300 mg/ day [4].…”

“…Allogeneic hematologic stem cell transplantation (HSCT) has been used with some success in four patients with relapsing PRCA or PRCA refractory to the conventional modalities [3][4][5][6]. Allogeneic HSCT has been claimed as the most effective procedure because it supplies a naïve immune system and provides a powerful graft-versus-autoimmunity (GVA) effect [7].…”