Treosulfan-based Conditioning in Paediatric Patients With Haematological Malignancies

Inclusion Criteria

• •1. Haematological malignant disease i.e. ALL, AML, MDS or JMML, indicated for allo-HSCT.
• •2. Indication for first allo-HSCT or second allo-HSCT due to disease relapse, graft failure, or secondary malignancy after previous HSCT.
• •3. Available matched sibling donor (MSD), matched family donor (MFD) or matched unrelated donor (MUD). For bone marrow (BM) and peripheral blood (PB) match is defined as 9/10 or 10/10 allele match after four digit typing in human leucocyte antigens (HLA)-A, B, C, DRB1 and DQB1.
• •4. Patients with ALL or AML in complete morphologic remission (blast counts \<5 % in BM) and patients with MDS or JMML with blast counts \< 20 % in BM at study entry.
• •5. Age at time of registration from 28 days to less than 18 years of age.
• •6. Lansky (patients aged \<16 years) or Karnofsky (patients aged ≥ 16 years) performance score of at least 70 %.
• •7. Written informed consent of the parents/ legal guardians and patient's assent/consent according to national regulations.
• •8. Females of child-bearing potential or male patients' partners with child-bearing potential must use a highly effective method of contraception (pearl index \< 1 %) such as complete sexual abstinence, combined oral contraceptive, hormone intrauterine contraceptive device (IUCD), vaginal hormone ring, transdermal contraceptive patch, contraceptive implant or depot contraceptive injection in combination with a second method of contraception like a condom or a cervical cap / diaphragm with spermicide or surgical sterilisation (vasectomy) in male patients or male partners during the study and at least 6 months thereafter.
• •9. Negative pregnancy test for females of child-bearing potential.