Przeszczepianie komórek krwiotwórczych ze zredukowanÄ toksycznościÄ i intensywnościÄ kondycjonowania u dzieci: rola treosulfanu

Hematopoietic stem cell transplantation (HSC T) is a procedure based on administration of hematopoietic stem cells from donor (autologous, allogeneic, syngeneic), regardless of source (bone marrow, peripheral blood, cord blood) to recipient after conditioning therapy in order to repopulate fully or partially recipient's hematopoietic system. Pretransplant conditioning therapy is based on high-dose chemotherapy or total body irradiation (TBI) and is aimed to obtain myeloablative, immunossupprssive and anticancer effect. Apart from myeloablation, the biological effect is the second important mechanism of allogeneic-HSC T; this effect is related to immunological reaction of transplanted donor's cells against recipient's residual leukemic cells (graft-versus-leukemia, GVL; graft-versus-tumor, GV T), but also against other host cells and organs (graft-versus-host, GV H). The cytotoxic agents most often used in pre-transplant condition therapy include busulfan, cyclophosphamide, melfalan and etoposide (I generation). With the introduction of fludarabine (II generation) to transplantology, the new possibilities has arisen, leading to HSC T with reduced intensity conditioning (RIC). Treosulfan is a relatively new compound, which is more and more often used in the hematopoietic stem cell transplantation setting. Anticancer, myeloablative and immunosuppressive properties of treosulfan, combined with its low toxicity and favorable pharmacokinetics have resulted in its high interest in transplantology and its potential use in condition regimens. Treosulfan has high safety and efficacy profile. These properties enable planning therapy for patients with non-malignant disorders qualified to hematopoietic stem cell transplantation.