Long-term follow-up of children conditioned with Treosulfan: German and Austrian experience

Bone Marrow Transplantation

Volumn 48, Issue 4, 2013, Pages 491-501

  Beier, R ^a   Schulz, A ^b   Honig M ^b   Eyrich, M ^c   Schlegel, P G ^c   Holter, W ^d   Stachel, K D ^e   Ehlert, K ^f   Greil, J ^g   Nurnberger W ^h   Wossmann W ^h   Bader, P ⁱ   Urban, C ^j   Muller I ^k,o   Suttorp, M ^l   Sauer, M ^a   Gruhn, B ^m   Meisel, R ⁿ   Zimmermann, M ^a   Sykora, K W ^a  

^a HANNOVER MEDICAL SCHOOL   (Germany)

^b UNIVERSITY OF ULM   (Germany)

^c UNIVERSITY OF WÜRZBURG   (Germany)

^d ST ANNA CHILDREN S HOSPITAL   (Austria)

^e UNIVERSITY HOSPITAL ERLANGEN   (Germany)

^f UNIVERSITY HOSPITAL MÜNSTER   (Germany)

^g UNIVERSITY HOSPITAL HEIDELBERG   (Germany)

^h UNIVERSITY HOSPITAL GIESSEN   (Germany)

ⁱ GOETHE UNIVERSITY   (Germany)

^j MEDICAL UNIVERSITY OF GRAZ   (Austria)

^k UNIVERSITY HOSPITAL TÜBINGEN   (Germany)

^l UNIVERSITY HOSPITAL CARL GUSTAV CARUS   (Germany)

^m JENA UNIVERSITY HOSPITAL   (Germany)

ⁿ HEINRICH HEINE UNIVERSITY   (Germany)

^o UNIVERSITY MEDICAL CENTER HAMBURG EPPENDORF   (Germany)

more..

Author keywords

children; haematopoietic SCT; inborn errors of metabolism; leukaemia; long term follow up; treosulfan

Indexed keywords

ALEMTUZUMAB; ANTIBODY; CD66 ANTIBODY; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; DEFIBROTIDE; FLUDARABINE; GRANULOCYTE COLONY STIMULATING FACTOR; MELPHALAN; METHOTREXATE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; OKT 3; RITUXIMAB; SILDENAFIL; THIOTEPA; THYMOCYTE ANTIBODY; TREOSULFAN; UNCLASSIFIED DRUG; YTTRIUM;

ADOLESCENT; ARTICLE; AUSTRIA; BONE MARROW TRANSPLANTATION; CANCER MORTALITY; CANCER SURVIVAL; CHILD; CONTROLLED STUDY; DIAPER DERMATITIS; DISEASE REGISTRY; ENGRAFTMENT; EVENT FREE SURVIVAL; FEMALE; FOLLOW UP; GASTROINTESTINAL TOXICITY; GERMANY; GRAFT VERSUS HOST REACTION; HEART RIGHT VENTRICLE HYPERTROPHY; HEMATOLOGIC DISEASE; HEMATOLOGIC MALIGNANCY; HIGH RISK PATIENT; HUMAN; HUMAN CELL; HYPERTENSION; IMMUNE DEFICIENCY; IMMUNOSUPPRESSIVE TREATMENT; LEUKEMIA; LEUKEMIA RELAPSE; LEUKEMIA REMISSION; LIVER VENOOCCLUSIVE DISEASE; LUNG TOXICITY; MAJOR CLINICAL STUDY; MALE; MALIGNANT NEOPLASTIC DISEASE; METABOLIC DISORDER; PERICARDITIS; PRIORITY JOURNAL; PULMONARY HYPERTENSION; RISK ASSESSMENT; SCORING SYSTEM; SEIZURE; SKIN TOXICITY; SKIN ULCER; SURVIVAL RATE; SURVIVAL TIME; VEIN OCCLUSION; WHOLE BODY RADIATION;

ADOLESCENT; ADULT; ANTINEOPLASTIC AGENTS, ALKYLATING; AUSTRIA; BONE MARROW TRANSPLANTATION; BUSULFAN; CHILD; CHILD, PRESCHOOL; COMMON VARIABLE IMMUNODEFICIENCY; DISEASE-FREE SURVIVAL; FEMALE; FOLLOW-UP STUDIES; GERMANY; HUMANS; INFANT; MALE; METABOLISM, INBORN ERRORS; MYELOABLATIVE AGONISTS; NEOPLASMS; REGISTRIES; RISK FACTORS; SURVIVAL RATE; TRANSPLANTATION CONDITIONING;

EID: 84876406371     PISSN: 02683369     EISSN: 14765365     Source Type: Journal    
DOI: 10.1038/bmt.2012.188     Document Type: Article

References (21)

1. Casper J, Knauf W, Kiefer T, Wolff D, Steiner B, Hammer U et al. Treosulfan and fludarabine: a new toxicity-reduced conditioning regimen for allogeneic hema-topoietic stem cell transplantation. Blood 2004; 103: 725-731.
2. Glowka FK, Romanski M, Wachowiak J. High-dose treosulfan in conditioning prior to hematopoietic stem cell transplantation. Expert. Opin Investig Drugs 2010; 19: 1275-1295.
3. Sauer M, Zeidler C, Meissner B, Rehe K, Hanke A, Welte K et al. Substitution of cyclophosphamide and busulfan by fludarabine, treosulfan and melphalan in a preparative regimen for children and adolescents with Shwachman-Diamond syndrome. Bone Marrow Transplant 2007; 39: 143-147.
4. Greystoke B, Bonanomi S, Carr TF, Gharib M, Khalid T, Coussons M et al. Treo-sulfan-containing regimens achieve high rates of engraftment associated with low transplant morbidity and mortality in children with non-malignant disease and significant co-morbidities. Br J Haematol 2008; 142: 257-262.
5. Cutting R, Mirelman A, Vora A. Treosulphan as an alternative to busulphan for myeloablative conditioning in paediatric allogeneic transplantation. Br J Haematol 2008; 143: 748-751.
6. Slatter MA, Rao K, Amrolia P, Flood T, Abinun M, Hambleton S et al. Treosulfan-based conditioning regimens for hematopoietic stem cell transplantation in children with primary immunodeficiency: United Kingdom experience. Blood 2011; 117: 4367-4375.
7. Wachowiak J, Sykora KW, Cornish J, Chybicka A, Kowalczyk JR, Gorczynska E et al. Treosulfan-based preparative regimens for allo-HSCT in childhood hematological malignancies: a retrospective study on behalf of the EBMT pediatric diseases working party. Bone Marrow Transplant 2011; 46: 1510-1518.
8. Matthes-Martin S, Potschger U, Bergmann K, Frommlet F, Brannath W, Bauer P et al. Risk-adjusted outcome measurement in pediatric allogeneic stem cell transplantation. Biol Blood Marrow Transplant 2008; 14: 335-343.
9. Kotlarz D, Beier R, Murugan D, Diestelhorst J, Jensen O et al. Loss of interleukin-10 signaling and infantile inflammatory bowel disease: implications for diagnosis and therapy. Gastroenterology 2012; 143: 347-355.
10. Wachowiak J, Grund G, Sykora KW, Wynn RF, Kowalczyk JR, Drabko K et al. Treosulfan-based conditioning regimen for allogeneic transplantation in children congenital non-malignant disorders-retrospective study. Bone Marrow Transplant 2008; 41: S80-S343.
11. Beier R, Schulz AS, Hoenig M, Schlegel G, Holter W, Stachel D et al. Treosulfan-based conditioning in children: retrospective analysis of the German and Austrian experience. Bone Marrow Transplant 2010; 45: S1-S11.
12. Poetschger U, Sykora KW, Zecca M, Veys P, Lankaster A, Cant A et al. Meta-analysis on treosulfan for conditioning in children and adolescents before haematopoietic stem cell transplantation. Bone Marrow Transplant 2012; 47: S1-S87.
13. Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J et al. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant 1995; 15: 825-828.
14. Filipovich AH, Weisdorf D, Pavletic S, Socie G, Wingard JR, Lee SJ et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant 2005; 11: 945-956.
15. Corbacioglu S, Greil J, Peters C, Wulffraat N, Laws HJ, Dilloo D et al. Defibrotide in the treatment of children with veno-occlusive disease (VOD): a retrospective multicentre study demonstrates therapeutic efficacy upon early intervention. Bone Marrow Transplant 2004; 33: 189-195.
16. Bernardo ME, Zecca M, Piras E, Vacca A, Giorgiani G, Cugno C et al. Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major. Br J Haematol 2008; 143: 548-551.
17. Nemecek ER, Guthrie KA, Sorror ML, Wood BL, Doney KC, Hilger RA et al. Conditioning with treosulfan and fludarabine followed by allogeneic hematopoietic cell transplantation for high-risk hematologic malignancies. Biol Blood Marrow Transplant 2011; 17: 341-350.
18. Holowiecki J, Giebel S, Wojnar J, Krawczyk-Kulis M, Markiewicz M, Holowiecka-Goral A et al. Treosulfan and fludarabine low-toxicity conditioning for allogeneic haematopoietic stem cell transplantation in chronic myeloid leukaemia. Br J Haematol 2008; 142: 284-292.
19. Hilgendorf I, Wolff D, Gromke T, Trenschel R, Elmaagacli AH, Pichlmeier U et al. Retrospective analysis of treosulfan-based conditioning in comparison with standard conditioning in patients with myelodysplastic syndrome. Bone Marrow Transplant 2011; 46: 502-509.
20. Koenigsmann M, Mohren M, Jentsch-Ullrich K, Franke A, Becker E, Heim M et al. High-dose treosulfan in patients with relapsed or refractory high-grade lymphoma receiving tandem autologous blood stem cell transplantation. Bone Marrow Transplant 2004; 34: 477-483.
21. Chemnitz JM, von Lilienfeld-Toal M, Holtick U, Theurich S, Shimabukuro-Vornhagen A, Krause A et al. Intermediate intensity conditioning regimen containing FLAMSA, treosulfan, cyclophosphamide, and ATG for allogeneic stem cell transplantation in elderly patients with relapsed or high-risk acute myeloid leukaemia. Ann Hematol 2012; 91: 47-55.