In the absence of a donor alternative, a stem cell transplantation consisting of two cord blood components originating from the haploidentical brother was performed in a 2-year-old girl with c-ALL, early CNS relapse and 7% of blast cells in the BM 14 days before transplantation.

The child's pneumonia as well as the septicemia subsided within a few days.

Notably, as well is the clearly shortened aplastic phase observed after this simultaneous CB cell component transplantation.

The patients T cell and NK cell reconstitution could be detected at day +37 with 330 CD3+ cells/microl and 40 CD56+ cells/microl, respectively.

The time to reach an absolute platelet count of 20 000 (50 000)/microl was 75 (103) days.

The disease-free survival now exceeds 1 year in complete remission without chronic GVHD or any other health problems.

These data show that the applicability of ex vivo expanded committed progenitors and LTC-IC, even in high risk leukemia at the time of transplantation, is feasible and can provide sufficient myeloid progenitors resulting in rapid engraftment able to clear bacterial pneumonia and sepsis.

In addition, accelerated hematopoietic reconstitution apparently served as a well functioning platform for definitive graft-versus-leukemia activity.

This transplantation of defined ex vivo generated components presents a feasible and generally applicable approach and may open a promising new avenue for cell therapy in malignant diseases.

Kogler, G. et al.Bone Marrow Transplant 1999 Aug;24(4):397-403.