Autologous peripheral blood stem cell transplantation and anti-B-cell directed immunotherapy for refractory auto-immune haemolytic anaemia

We report the clinical course of a 6.5-year-old boy with refractory auto-im mune haemolytic anaemia. Due to failure of conventional immunosuppressive t herapy, an autologous peripheral blood stem cell transplantation was perfor med. The conditioning regimen consisted of cyclophosphamide and anti-thymoc yte globulin. The patient was reinfused with 2.6 x 10(6) CD34 positive sele cted, B- and T-cell-depleted peripheral blood stem cells per kg body weight . He showed a partial response with a reduced demand for red blood cell tra nsfusions. However, due to persistence of the haemolytic process he was sta rted on rituximab therapy on day +40 post-transplant. Following two doses o f rituximab, the patient improved rapidly and developed a sustained complet e response. After 10 months, haemolysis recurred and responded again to rit uximab therapy without the necessity for red blood cell transfusions. 15 mo nths after initial antibody treatment, however, the patient developed a sec ond relapse which was now refractory to rituximab therapy although CD20 + B -lymphocytes were cleared from the peripheral blood. Conclusion Our case report suggests that rituximab and autologous periphera l blood stem cell transplantation are important though not curative element s in the treatment of patients with severe auto-immune haemolytic anaemia w ho are refractory to conventional immunosuppressive therapy.