Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major

British Journal of Haematology

Volumn 143, Issue 4, 2008, Pages 548-551

  Bernardo, Maria Ester ^a,b   Zecca, Marco ^b   Piras, Eugenia ^c   Vacca, Adriana ^c   Giorgiani, Giovanna ^b   Cugno, Chiara ^b   Caocci, Giovanni ^c   Comoli, Patrizia ^b   Mastronuzzi, Angela ^b   Merli, Pietro ^b   La Nasa, Giorgio ^c   Locatelli, Franco ^b  

^a FONDAZIONE IRCCS POLICLINICO SAN MATTEO   (Italy)

^b UNIVERSITY OF PAVIA   (Italy)

^c Binaghi Hospital   (Italy)

Author keywords

Conditioning regimen; Haematopoietic stem cell transplantation; Thalassaemia major; Toxicity; Treosulfan

Indexed keywords

FLUDARABINE; THIOTEPA; TREOSULFAN;

ADOLESCENT; ADULT; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; CARDIOTOXICITY; CHILD; CLINICAL ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DISEASE FREE SURVIVAL; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; FEMALE; GRAFT FAILURE; GRAFT VERSUS HOST REACTION; HUMAN; INFANT; LIVER TOXICITY; LUNG TOXICITY; MALE; MORTALITY; MUCOSA INFLAMMATION; MYELOABLATIVE CONDITIONING; NEUROTOXICITY; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; THALASSEMIA MAJOR; TREATMENT OUTCOME;

ADOLESCENT; ADULT; BETA-THALASSEMIA; BUSULFAN; CHILD; CHILD, PRESCHOOL; DRUG THERAPY, COMBINATION; FEMALE; GRAFT REJECTION; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INFANT; MALE; MYELOABLATIVE AGONISTS; THIOTEPA; TRANSPLANTATION CONDITIONING; TREATMENT OUTCOME; VIDARABINE; YOUNG ADULT;

EID: 54849419695     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/j.1365-2141.2008.07385.x     Document Type: Article

References (12)

1. Bearman, S.I., Appelbaum, F.R., Buckner, C.D., Petersen, F.B., Fisher, L.D., Clift, R.A. Thomas, E.D. (1988) Regimen related toxicity in patients undergoing bone marrow transplantation. Journal of Clinical Oncology, 6, 1562 1568.
2. Beelen, D.W., Trenschel, R., Casper, J., Freund, M., Hilger, R.A., Scheulen, M.E., Basara, N., Fauser, A.A., Hertenstein, B., Mylius, H.A., Baumgart, J., Pichlmeier, U., Hahn, J.R. Holler, E. (2005) Dose-escalated treosulfan in combination with cyclophosphamide as a new preparative regimen for allogeneic haematopoietic stem cell transplantation in patients with an increased risk for regimen-related complications. Bone Marrow Transplantation, 35, 233 241.
3. Casper, J., Knauf, W., Kiefer, T., Wolff, D., Steiner, B., Hammer, U., Wegener, R., Kleine, H.D., Wilhelm, S., Knopp, A., Hartung, G., Dölken, G. Freund, M. (2004) Treosulfan and fludarabine: a new toxicity-reduced conditioning regimen for allogeneic haematopoietic stem cell transplantation. Blood, 103, 725 731.
4. Glucksberg, H., Storb, R., Fefer, A., Buckner, C.D., Neiman, P.E., Clift, R.A., Lerner, K.G. Thomas, E.D. (1974) Clinical manifestations of graft-versus-host disease in human recipients of marrow from HLA-matched sibling donors. Transplantation, 18, 295 304.
5. Hilger, R.A., Harstrick, A., Eberhardt, W., Oberhoff, C., Skorzec, M., Baumgart, J., Seeber, S. Scheulen, M.E. (1998) Clinical pharmacokinetics of intravenous treosulfan in patients with advanced solid tumors. Cancer Chemotherapy Pharmacology, 42, 99 104.
6. Iannone, R., Casella, J.F., Fuchs, E.J., Chen, A.R., Jones, R.J., Woolfrey, A., Amylon, M., Sullivan, K.M., Storb, R.F. Walters, M.C. (2003) Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and β-thalassemia. Biology of Blood and Marrow Transplantation, 9, 519 528.
7. La Nasa, G., Caocci, G., Argiolu, F., Giardini, C., Locatelli, F., Vacca, A., Orofino, M.G., Piras, E., Addari, M.C., Ledda, A. Contu, L. (2005) Unrelated donor bone marrow transplantation in adult patients with thalassemia. Bone Marrow Transplantation, 36, 971 975.
8. Locatelli, F., Rocha, V., Reed, W., Bernaudin, F., Ertem, M., Grafakos, S., Brichard, B., Li, X., Nagler, A., Giorgiani, G., Haut, P.R., Brochstein, J.A., Nugent, D.J., Blatt, J., Woodard, P., Kurtzberg, J., Rubin, C.M., Miniero, R., Lutz, P., Raja, T., Roberts, I., Will, A.M., Yaniv, I., Vermylen, C., Tannoia, N., Garnier, F., Ionescu, I., Walters, M.C., Lubin, B.H. Gluckman, E. for the Eurocord Transplant Group CBT (2003) Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood, 101, 2137 2143.
9. Lucarelli, G., Galimberti, M., Polchi, P., Angelucci, E., Baronciani, D., Giardini, C., Andreani, M., Agostinelli, F., Albertini, F. Clift, R.A. (1993) Marrow transplantation in patients with thalassemia responsive to iron chelation therapy. The New England Journal of Medicine, 329, 840 844.
10. Lucarelli, G., Clift, R.A., Galimberti, M., Polchi, P., Angelucci, E., Baronciani, D., Giardini, C., Andreani, M., Manna, M., Nesci, S., Agostinelli, F., Rapa, S., Ripalti, M. Albertini, F. (1996) Marrow transplantation for patients with thalassemia: results in class 3 patients. Blood, 87, 2082 2088.
11. Munkelt, D., Koehl, U., Kloess, S., Zimmermann, S.Y., Kalaäoui, R.E., Wehner, S., Schwabe, D., Lehrnbecher, T., Schubert, R., Kreuter, J., Klingebiel, T. Esser, R. (2008) Cytotoxic effects of treosulfan and busulfan against leukemic cells of pediatric patients. Cancer Chemotherapy and Pharmacology, 62, 821 830.
12. Ploemacher, R.E., Johnson, K.W., Rombouts, E.J., Etienne, K., Westerhof, G.R., Baumgart, J., White-Scharf, M.E. Down, J.D. (2004) Addition of treosulfan to a nonmyeloablative conditioning regimen results in enhanced chimerism and immunologic tolerance in an experimental allogeneic bone marrow transplant model. Biology of Blood and Marrow Transplantation, 10, 236 245.