Nonmyeloablative Conditioning Followed by Allogeneic Marrow Grafts for Treatment of Primary Immune Deficiency Disorders: Preliminary Results of a Phase II Study

L. Burroughs, T. Torgerson, B. Storer, W. Leisenring, E. Nemecek, H. Frangoul, M.C. Walters, A. Scharenberg, D.J. Rawlings, S. Skoda-Smith, H. Ochs, R. Storb (+1 others)
2011 Biology of Blood and Marrow Transplantation  
CB, and 3-4/6 unrelated CB. Tacrolimus and mycophenolate mofetil were used as GVHD prophylaxis (Bhatia/Cairo et al, BBMT 2009) and phenytoin or levetiracetam as seizure prophylaxis for 180 days post-SCT. Organ function testing (MRI/MRA, ECHO, and PFT) was done pre-SCT and 1 and 2 yrs post-SCT in evaluable patients. The median follow up is 2.3 yrs (0.6-6.1 yrs). The OS, EFS, and median 1 and 2 year donor chimerism (WB/CD71) are 83%, 78%, 93/90% and 94/95%, respectively (Bhatia/Cairo et al, ASH
more » ... 10). Of the 15 evaluable patients, 9 and 7 patients had a repeat MRI at 1 and 2 yrs, respectively. All had stable or improved MRI with the exception of 1 pt who had an embolic CVA on MRI 7 months post-SCT which resolved one month later. Sixteen of 18 patients had pre-SCT ECHO and of those, 3 were noted to have mild to moderate tricuspid regurgitation (TR). Of those three patients, two were evaluable at 1 and 2 yrs post-SCT, and were noted to have improvement in degree of TR, which was noted to be trivial in one, and trivial to mild in the other. Of evaluable patients at 1 (n 5 9) and 2 yrs (n 5 7) post-SCT, all were noted to have stable or improving TR with no patient demonstrating evidence of pulmonary hypertension. All patients greater than 8 yrs of age underwent PFTs. 11 patients had pre-SCT PFTs with 2/11 having restrictive disease and 1/11 having obstructive disease. Of the nine patients who were evaluable for comparison before and after SCT, the mean predicted FVC changed from 90 6 23% to 93.5 6 16% and the mean predicted FEV1 changed from 87 6 29% to 90 6 18% after transplantation. In conclusion, RTC SCT effectively promoted stable donor engraftment in SCD pts and was associated with stable or improved organ stability function at 2 years post-SCT.
doi:10.1016/j.bbmt.2010.12.088 fatcat:7pj3weq4dzdjnp5cxqpi3ywsim