Table. 2.

Table. 2.

Selected reports on hematopoietic stem cell transplantation after treosulfan-based conditioning in pediatric patients with non-malignant disease

Study/author Year N Diagnosis (n) Main conditioning regimens VOD
(n or %)
Mixed chimerism GVHD (n or %) TRM
(n or %)
Survival Ref

Acute (gr) Chronic
Lehmberg 2014 19 HLH (19) Treo, Flu, Alem±TT 1 53% 6% (3) 0% 0% 100% 45
Burroughs 2014 31 PID (13), HLH (6), DBA (3), SDS (3), SCD (4), PNH (2) Treo, Flu±TT 0% 29% (CD3+) 10% (3-4) 21% 10% 90% 36
Dinur-Schejter 2015 44 PID (27), hematologic (9), metabolic (9) Treo, Flu±TT NS 28% 27% (3-4) 19% 12 71% 37
EBMT/Slatter 2015 316 Inherited disorders (188), hemoglobinopathy (70), histiocytic disorder (32), BMF (24), others (2) Treo, Flu±TT Treo, Cy 0% (≥grade 3) NS 10% (3-4) NS 51 91% for thalassemia, 100% for SCD 38
Morillo-Gutierrez 2016 70 CGD (70) Treo, Flu±TT±ATG or Alem 0% 25% (CD3+), 20% (CD15+) 12% (3-4) 13% 6 91.4% 39
FHCRC/Burroughs 2017 14 SDS (3), DBA (4), PNH (4), others (3) Treo, Flu 0% 7% 1 (4) 2 1 13 alive 40
Slatter 2018 160 SCID (39), WAS (20), CGD (17), HLH (18), others (66) Treo, Flu, Alem 0% Various 9% (3-4) 24 27 83% 41

HLH, hemophagocytic lymphohistiocytosis; PID, primary immunodeficiency; DBA, Diamond-Blackfan anemia; SDS, Shwachman-Diamond syndrome; SCD, sickle cell disease; PNH, paroxysmal nocturnal hemoglobinuria; BMF, bone marrow failure; CGD, chronic granulomatous disease; SCID, severe combined immunodeficiency; WAS, Wiskott-Aldrich syndrome; VOD, veno-occlusive disease; TRM, transplant-related mortality; Treo, treosulfan 36 or 42 g/m2; Flu, fludarabine 150-180 mg/m2; TT, thiotepa 7-10 mg/kg; ATG, antithymocyte globulin; Alem, alemtuzumab; Cy, cyclophosphamide 200 mg/kg; NS, not specified; Ref, reference.

Clin Pediatr Hematol Oncol 2021;28:28-38
© 2021 Clin Pediatr Hematol Oncol