Autologous peripheral blood stem cell transplantation and anti-B-cell directed immunotherapy for refractory auto-immune haemolytic anaemia.
ABSTRACT We report the clinical course of a 6.5-year-old boy with refractory auto-immune haemolytic anaemia. Due to failure of conventional immunosuppressive therapy, an autologous peripheral blood stem cell transplantation was performed. The conditioning regimen consisted of cyclophosphamide and anti-thymocyte globulin. The patient was reinfused with 2.6 x 10(6) CD34 positive selected, 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 transfusions. However, due to persistence of the haemolytic process he was started on rituximab therapy on day +40 post-transplant. Following two doses of rituximab, the patient improved rapidly and developed a sustained complete response. After 10 months, haemolysis recurred and responded again to rituximab therapy without the necessity for red blood cell transfusions. 15 months after initial antibody treatment, however, the patient developed a second 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 peripheral blood stem cell transplantation are important though not curative elements in the treatment of patients with severe auto-immune haemolytic anaemia who are refractory to conventional immunosuppressive therapy.
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ABSTRACT: Stem cells are a relevant source of information about cellular differentiation, molecular processes and tissue homeostasis, but also one of the most putative biological tools to treat degenerative diseases. This review focuses on human stem cells clinical and experimental applications. Our aim is to take a correct view of the available stem cell subtypes and their rational use in the medical area, with a specific focus on their therapeutic benefits and side effects. We have reviewed the main clinical trials dividing them basing on their clinical applications, and taking into account the ethical issue associated with the stem cell therapy. METHODS: We have searched Pubmed/Medline for clinical trials, involving the use of human stem cells, using the key words "stem cells" combined with the key words "transplantation", "pathology", "guidelines", "properties" and "risks". All the relevant clinical trials have been included. The results have been divided into different categories, basing on the way stem cells have been employed in different pathological conditions.Journal of Experimental & Clinical Cancer Research 01/2011; 30:9. · 3.07 Impact Factor
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ABSTRACT: There has been a remarkable history in the treatment of patients with autoimmune disease in the last century. Prior to the development of newer NSAIDs and corticosteroids, the care of patients with autoimmune disease was limited to aspirin and generally homeopathic therapies such as paraffin. In the last 30 years, the introduction and acceptance of cytotoxic drugs such as methotrexate and cyclophosphamide have greatly advanced the treatment of patients with severe autoimmune diseases. However, the use and dose escalation of cytotoxic agents in severely ill patients is limited by toxicity and the potential for secondary malignancies that correlate with cumulative lifetime dosing. As hematopoietic stem cell transplant grew to become an established procedure for certain malignancies, reports of remission of coexistent autoimmune diseases began to emerge. Animal data subsequently supported a role for hematopoietic stem cell transplants for the primary indication of autoimmune diseases. On the basis of these reports, clinical trials of hematopoietic stem cell transplants for the primary indication of autoimmune disease were initiated in the late 1990s. We review the data from a decade of experience that has now accumulated for this novel approach to the management of autoimmunity.Journal of Autoimmunity 06/2008; 30(3):180-96. · 8.15 Impact Factor
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ABSTRACT: Autoimmune hemolytic anemia (AIHA) is an immune mediated destruction of erythrocytes, which has a good prognosis in children. It is known to have chronic, remitting or relapsing course, especially in infants and adolescents. Treatment of refractory or relapsing AIHA is a challenge as the other aim of the treatment is to avoid prolonged exposure to steroids or other immunosuppressants in small children. Rituximab is used in patients who are non-responsive to conventional treatment such as steroids, intravenous immunoglobulins and transfusion therapy. It has varying therapeutic success rate. We report a case of AIHA in a 4-month-old infant who had ill-sustained response to conventional therapy, but responded to rituximab.Indian Journal of Pharmacology 01/2013; 45(5):526-527. · 0.58 Impact Factor