Treosulfan/fludarabine: a new conditioning regimen in allogeneic transplantation

Ann Hematol. 2004:83 Suppl 1:S70-1. doi: 10.1007/s00277-004-0850-2.

Abstract

Recently, the water-soluble bifunctional alkylating agent treosulfan demonstrated broad stem cell toxicity, immunosuppressive as well as antileukemic activity. Due to its well known low non-hematologic toxicity profile, treosulfan was considered an alternative agent for conditioning prior to allogeneic transplantation. A first clinical study, combining 3 x 10 g/m2 of treosulfan with 5 x 30 mg/m2 of fludarabine, demonstrated the feasibility of this conditioning. A fast, reliable and complete development of the donor hematopoiesis was evident as well as a low non-hematologic toxicity, transplantation-related mortality and relapse rate. In a second study treosulfan was escalated from 3 x 10 to 3 x 12 and 3 x 14 g/m2. In this protocol, 55 pts (patients) not amenable to standard conditioning suffering from various hematological malignancies were included. Complete donor chimerism was reached by day 28 in 80% of the pts. So far, 8 pts (11%) died without disease progression and 11 pts (20%) relapsed. Treosulfan was very well tolerated. Especially no hepatic VOD, severe cardiac or pulmonary toxicity was noted. Acute GvHD (degrees 11-IV) occurred in 44% and chronic GvHD in 45% of pts. Considering the poor prognosis of these study populations, treosulfan-based conditioning is considered to be safe and efficient. New phase 11 clinical protocols in AML and MDS will be initiated.

Publication types

  • Clinical Trial
  • Controlled Clinical Trial

MeSH terms

  • Busulfan / analogs & derivatives*
  • Busulfan / therapeutic use*
  • Humans
  • Leukemia / classification
  • Leukemia / therapy*
  • Leukemia, Myeloid, Acute / drug therapy
  • Leukocyte Transfusion*
  • Lymphoma / classification
  • Lymphoma / therapy*
  • Myelodysplastic Syndromes / drug therapy
  • Stem Cell Transplantation*
  • Transplantation Conditioning / methods*
  • Transplantation, Homologous / immunology*
  • Transplantation, Homologous / methods*
  • Vidarabine / analogs & derivatives*
  • Vidarabine / therapeutic use*

Substances

  • treosulfan
  • Vidarabine
  • Busulfan
  • fludarabine