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Blood, 15 August 2004, Vol. 104, No. 4, pp. 1201-1203.
Prepublished online as a Blood First Edition Paper on March 23, 2004; DOI 10.1182/blood-2003-08-2800.


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TRANSPLANTATION

New approach for bone marrow transplantation in patients with class 3 thalassemia aged younger than 17 years

Pietro Sodani, David Gaziev, Paola Polchi, Buket Erer, Claudio Giardini, Emanuele Angelucci, Donatella Baronciani, Marco Andreani, Marisa Manna, Sonia Nesci, Barbarella Lucarelli, Reginald A. Clift, and Guido Lucarelli

From the Unità Operativa Ematologia e Centro Trapianto di Midollo Osseo di Muraglia, Azienda Ospedale S. Salvatore di Pesaro, Pesaro, Italy; the Clinical Division, Fred Hutchinson Cancer Research Center, Seattle, WA; and the Mediterranean Institute of Hematology, Rome, Italy.

When prepared for transplantation with busulfan (BU) 14 mg/kg and cyclophosphamide (CY) 120 to 160 mg/kg, patients with thalassemia in risk class 3, aged younger than 17 years, who receive transplants from HLA-identical donors, had a 30% incidence of transplant rejection with recurrence of thalassemia. This, relatively poor, outcome was ascribed to insufficient immune suppression or to inadequate eradication of the thalassemic marrow, or both. In an attempt to enhance both immune suppression and eradication of the thalassemic clones, hydroxyurea, azathioprine, and fludarabine were added to the BU and CY. This regimen, called protocol 26, was applied to 33 consecutive patients with class 3 thalassemia aged younger than 17 years and was well tolerated with 93% survival. The incidence of recurrent thalassemia after the transplantation decreased from 30% to 8%. (Blood. 2004;104:1201-1203)


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