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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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Submitted August 14, 2003
Accepted February 25, 2004
A new approach for bone marrow transplantation in class 3 thalassemic patients aged less than 17 years
Pietro Sodani, Djavid Gaziev, Paola Polchi, Buret Erer, Claudio Giardini, Emanuele Angelucci, Donatella Baronciani, Marco Andreani, Marisa Manna, Sonia Nesci, Barbarella Lucarelli, Reginald A Clift*, and Guido Lucarelli
Azienda Ospedale S. Salvatore di Pesaro, U.O. Ematologia e Centro Trapianto di Midollo Osseo di Muraglia, Pesaro, Italy
Clinical Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA
Mediterranean Institute of Hematology, Rome, Italy
* Corresponding author; email: rclift{at}fhcrc.org.
When prepared for transplantation with busulfan (BU) 14 mg/kg and cyclophosphamide (CY) 120 - 160 mg/kg, thalassemic patients in risk class 3, aged younger than 17 years, transplanted 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 class 3 thalassemic patients aged less than 17 years and was well tolerated with 93% survival. The incidence of recurrent thalassemia after the transplant decreased from 30 to 8 percent.

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