Myeloablative Transplantation Using Either Cord Blood or Bone Marrow Leads to Immune Recovery, High Long-Term Donor Chimerism and Excellent Survival in Chronic Granulomatous Disease
09/2012
Journal Article
Authors:
Tewari, P.;
Martin, P.L.;
Mendizabal, A.;
Parikh, S.H.;
Page, K.M.;
Driscoll, T.A.;
Malech, H.L.;
Kurtzberg, J.;
Prasad, V.K.
Secondary:
Biol Blood Marrow Transplant
Volume:
18
Pagination:
1368-1377
URL:
http://www.ncbi.nlm.nih.gov/pubmed/22326631
Keywords:
Chronic granulomatous disease; Cord blood; Myeloablative
Abstract:
{The curative potential of hematopoietic stem cell transplantation in patients with chronic granulomatous disease depends on availability of a suitable donor, successful donor engraftment, and maintenance of long-term donor chimerism. Twelve consecutive children (median age, 59.5 months; range, 8-140 months) with severe chronic granulomatous disease (serious bacterial/fungal infections pretransplantation; median, 3; range, 2-9) received myeloablative hematopoietic stem cell transplantation using sibling bone marrow ([SibBM]; n = 5), unrelated cord blood (UCB; n = 6), and sibling cord blood (n = 1) at our center between 1997 and 2010. SibBM and sibling cord blood were HLA matched at 6/6, whereas UCB were 5/6 (n = 5) or 6/6 (n = 1). Recipients of SibBM were conditioned with busulfan and cyclophosphamide ± anti-thymocyte globulin (ATG), whereas 6 of 7 cord blood recipients received fludarabine/busulfan/cyclophosphamide/ATG. Seven patients received granulocyte-colony stimulating factor-mobilized granulocyte transfusions from directed donors. The first 2 UCB recipients had primary graft failure but successfully underwent retransplantation with UCB. Highest acute graft-versus-host disease was grade III (n = 1). Extensive chronic graft-vs-host disease developed in 3 patients. All patients are alive with median follow-up of 70.5 months (range, 12-167 months) with high donor chimerism (>98%
