BACKGROUND AND AIMS: Autologous haematopoietic stem cell transplantation (HSCT) is considered a salvage therapy for patients with refractory immune-mediated diseases. Syngeneic HSCT may be an alternative to autologous HSCT, with potential advantages in terms of safety and efficacy. METHODS: A patient with severe Crohn's disease refractory to available medical therapies underwent a syngeneic HSCT from her identical twin sister. Cyclophosphamide and rabbit anti-thymocyte globulin were administered for conditioning. RESULTS: After transplant, the patient presented successful engraftment, complete haematological and immunological reconstitution, and no severe complications. The patient achieved complete remission after transplant which is sustained 4 years after transplantation without any active treatment for Crohn's disease. CONCLUSIONS: Patients with refractory immune-mediated diseases who have an identical disease-free twin may benefit from a syngeneic HSCT.
BACKGROUND AND AIMS: Autologous haematopoietic stem cell transplantation (HSCT) is considered a salvage therapy for patients with refractory immune-mediated diseases. Syngeneic HSCT may be an alternative to autologous HSCT, with potential advantages in terms of safety and efficacy. METHODS: A patient with severe Crohn's disease refractory to available medical therapies underwent a syngeneic HSCT from her identical twin sister. Cyclophosphamide and rabbit anti-thymocyte globulin were administered for conditioning. RESULTS: After transplant, the patient presented successful engraftment, complete haematological and immunological reconstitution, and no severe complications. The patient achieved complete remission after transplant which is sustained 4 years after transplantation without any active treatment for Crohn's disease. CONCLUSIONS:Patients with refractory immune-mediated diseases who have an identical disease-free twin may benefit from a syngeneic HSCT.
Authors: Philipp Schreiner; Markus F Neurath; Siew C Ng; Emad M El-Omar; Ala I Sharara; Taku Kobayashi; Tadakazu Hisamatsu; Toshifumi Hibi; Gerhard Rogler Journal: Inflamm Intest Dis Date: 2019-07-09