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

被引：54

作者：

Bernardo, Maria Ester ^{[1
]}

Zecca, Marco ^{[1
]}

Piras, Eugenia ^{[2
]}

Vacca, Adriana ^{[2
]}

Giorgiani, Giovanna ^{[1
]}

Cugno, Chiara ^{[1
]}

Caocci, Giovanni ^{[2
]}

Comoli, Patrizia ^{[1
]}

Mastronuzzi, Angela ^{[1
]}

Merli, Pietro ^{[1
]}

La Nasa, Giorgio ^{[2
]}

Locatelli, Franco ^{[1
]}

机构：

[1] Univ Pavia, Fdn IRCCS Policlin San Matteo, Oncoematol Pediat, I-27100 Pavia, Italy

[2] Osped R Binaghi, Unita Operat Ctr Trapianti Midollo Osseo, Cagliari, Italy

来源：

BRITISH JOURNAL OF HAEMATOLOGY | 2008年 / 143卷 / 04期

关键词：

haematopoietic stem cell transplantation; treosulfan; conditioning regimen; thalassaemia major; toxicity;

D O I：

10.1111/j.1365-2141.2008.07385.x

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

The safety and efficacy of a preparation with treosulfan/thiotepa/fludarabine were explored in 20 thalassaemia patients given allogeneic marrow transplantation. Seventeen patients were transplanted from unrelated donors after receiving anti-thymocyte globulin. The regimen was well tolerated. Two patients experienced secondary graft failure; one died of acute graft-versus-host disease. Cumulative incidence (95% confidence interval, CI) of transplantation-related mortality and graft failure was 5% (95% CI, 0-34%) and 11% (95% CI, 3-43%), respectively. Two-year probability of survival and thalassaemia-free survival was 95% (95% CI, 85-100%) and 85% (95% CI, 66-100%), respectively. This regimen might find elective application in patients at high risk of developing life-threatening complications.

引用

页码：548 / 551

页数：4

共 12 条

[1] REGIMEN-RELATED TOXICITY IN PATIENTS UNDERGOING BONE-MARROW TRANSPLANTATION [J].

BEARMAN, SI ;

APPELBAUM, FR ;

BUCKNER, CD ;

PETERSEN, FB ;

FISHER, LD ;

CLIFT, RA ;

THOMAS, ED .

JOURNAL OF CLINICAL ONCOLOGY, 1988, 6 (10) :1562-1568

[2] Dose-escalated treosulphan 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 [J].

Beelen, DW ;

Trenschel, R ;

Casper, J ;

Freund, M ;

Hilger, RA ;

Scheulen, ME ;

Basara, N ;

Fauser, AA ;

Hertenstein, B ;

Mylius, HA ;

Baumgart, J ;

Pichlmeier, U ;

Hahn, JR ;

Holler, E .

BONE MARROW TRANSPLANTATION, 2005, 35 (03) :233-241

[3] Treosulfan and fludarabine:: a new toxicity-reduced conditioning regimen for allogeneic hematopoietic stem cell transplantation [J].

Casper, J ;

Knauf, W ;

Kiefer, T ;

Wolff, D ;

Steiner, B ;

Hammer, U ;

Wegener, R ;

Kleine, HD ;

Wilhelm, S ;

Knopp, A ;

Hartung, G ;

Dölken, G ;

Freund, M .

BLOOD, 2004, 103 (02) :725-731

[4] CLINICAL MANIFESTATIONS OF GRAFT VERSUS HOST DISEASE IN HUMAN RECIPIENTS OF MARROW FROM HL-A-MATCHED SIBLING DONORS [J].

GLUCKSBERG, H ;

STORB, R ;

FEFER, A ;

BUCKNER, CD ;

NEIMAN, PE ;

CLIFT, RA ;

LERNER, KG ;

THOMAS, ED .

TRANSPLANTATION, 1974, 18 (04) :295-304

[5] Clinical pharmacokinetics of intravenous treosulfan in patients with advanced solid tumors [J].

Hilger, RA ;

Harstrick, A ;

Eberhardt, W ;

Oberhoff, C ;

Skorzec, M ;

Baumgart, J ;

Seeber, S ;

Scheulen, ME .

CANCER CHEMOTHERAPY AND PHARMACOLOGY, 1998, 42 (02) :99-104

[6] Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and β-thalassemia [J].

Iannone, R ;

Casella, JE ;

Fuchs, EJ ;

Chen, AR ;

Jones, RJ ;

Woolfrey, A ;

Amylon, M ;

Sullivan, KM ;

Storb, RF ;

Walters, MC .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2003, 9 (08) :519-528

[7] Unrelated donor stem cell transplantation in adult patients with thalassemia [J].

La Nasa, G ;

Caocci, G ;

Argiolu, F ;

Giardini, C ;

Locatelli, F ;

Vacca, A ;

Orofino, MG ;

Piras, E ;

Addari, MC ;

Ledda, A ;

Contu, L .

BONE MARROW TRANSPLANTATION, 2005, 36 (11) :971-975

[8] Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease [J].

Locatelli, F ;

Rocha, V ;

Reed, W ;

Bernaudin, F ;

Ertem, M ;

Grafakos, S ;

Brichard, B ;

Li, XX ;

Nagler, A ;

Giorgiani, G ;

Haut, PR ;

Brochstein, JA ;

Nugent, DJ ;

Blatt, J ;

Woodard, P ;

Kurtzberg, J ;

Rubin, CM ;

Miniero, R ;

Lutz, P ;

Raja, T ;

Roberts, I ;

Will, AM ;

Yaniv, I ;

Vermylen, C ;

Tannoia, N ;

Garnier, F ;

Ionescu, I ;

Walters, MC ;

Lubin, BH ;

Gluckman, E .

BLOOD, 2003, 101 (06) :2137-2143

[9] MARROW TRANSPLANTATION IN PATIENTS WITH THALASSEMIA RESPONSIVE TO IRON CHELATION-THERAPY [J].

LUCARELLI, G ;

GALIMBERTI, M ;

POLCHI, P ;

ANGELUCCI, E ;

BARONCIANI, D ;

GIARDINI, C ;

ANDREANI, M ;

AGOSTINELLI, F ;

ALBERTINI, F ;

CLIFT, RA .

NEW ENGLAND JOURNAL OF MEDICINE, 1993, 329 (12) :840-844

[10] Marrow transplantation for patients with thalassemia: Results in class 3 patients [J].

Lucarelli, G ;

Clift, RA ;

Galimberti, M ;

Polchi, P ;

Angelucci, E ;

Baronciani, D ;

Giardini, C ;

Andreani, M ;

Manna, M ;

Nesci, S ;

Agostinelli, F ;

Rapa, S ;

Ripalti, M ;

Albertini, F .

BLOOD, 1996, 87 (05) :2082-2088

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