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Second BMT for thalassemia major using CY post transplant

Bone Marrow Transplantation volume 49, pages 845–846 (2014)Cite this article

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Rejection after a matched related BMT for thalassemia major (TM) may occur in 5–30% of BMTs.1, 2, 3 Second BMT for TM using marrow from the original donor with aggressive conditioning regimens has been successfully performed at a cost of substantial mortality and morbidity with OS rates of 70–80%.4,5

On the basis of reports on the effectiveness of high-dose CY, 50 mg/kg/day on days +3 and +4 (PTCy) after unmanipulated BMT as a safe and potent GVHD/rejection prophylaxis,6,7 we chose this strategy for two patients who rejected their first compatible sibling graft. The second BMT from the same donor was performed using the same conditioning drugs, but shifting a proportion of the CY to days +3 and +4 with no additional immunosuppression. The first graft had the following conditioning regimen: oral BU 3.5 mg/kg/day in four divided doses on days −10 to −7 (total dose 14 mg/kg), thiotepa 10 mg/kg in two divided doses on day −6 and CY 50 mg/kg/day once daily on days −5 to −2 (total dose 200 mg/kg) followed by the infusion of freshly harvested HLA-compatible BM on day 0. GVHD prophylaxis consisted of CsA starting at 5 mg/kg i.v. from days −2 to +5 then 3 mg/kg i.v. from days +6 to +22 followed by 10 mg/kg/day in two daily oral doses for up to day +90 after which it was tapered by −5%/week and discontinued at 8–9 months post BMT. A short MTX course of 10 mg/m2 i.v. on days +1 (24 h after marrow infusion), +3 and +6, with folic acid rescue at 24 h after each MTX using three doses of 10 mg/m2 i.v. at 8-h intervals (that is, at hours +24 h +32 h and +40 h). Methylprednisolone at 0.5 mg/kg/day i.v. from day −1 to +30 and tapered by −1/3 every 5 days over 15 days and stopped on day +45 was also used (Lucarelli's protocol 6.1).8

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Acknowledgements

This work was partially supported by Fondazione Monte dei Paschi di Siena, Siena, Italy and by the Cure2Children Foundation, Florence, Italy.

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Authors and Affiliations

  1. Pakistan Institute of Medical Sciences (PIMS), Islamabad, Pakistan

    N Yaqub, F Itrat, A Khan & S K Gilani

  2. Cure2Children Foundation, Florence, Italy

    S Khalid, M E Missiry, M H Hussain, C Uderzo & L Faulkner

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  1. N Yaqub
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  2. S Khalid
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  3. F Itrat
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  4. A Khan
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  6. M E Missiry
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  7. M H Hussain
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  8. C Uderzo
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  9. L Faulkner
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Correspondence to L Faulkner.

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Yaqub, N., Khalid, S., Itrat, F. et al. Second BMT for thalassemia major using CY post transplant. Bone Marrow Transplant 49, 845–846 (2014). https://doi.org/10.1038/bmt.2014.36

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