PubMed · 10148864
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F M Fellin. 1993. Dealing with hematologic problems before surgery.. https://pubmed.ncbi.nlm.nih.gov/10148864/
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This case report describes for the first time the use of a recombinant human enzyme deoxyribonuclease (rhDNase) containing solution for the processing of a thawed umbilical cord blood (UCB) unit prior to successful transplantation to avoid cell losses by clotting phenomena. A 6-year-old boy received an unrelated 2/6 HLA antigen mismatched UCB transplant for high-risk Burkitt type acute lymphoblastic leukemia. The UCB unit was provided as a volume-reduced sample after buffy coat separation with a final volume of 36 ml. To avoid the loss of nucleated cells due to cell clumping during thawing procedure cells were washed with a solution containing the rhDNase. No visible clotting of the resuspended unit occurred, and the patient was transplanted with 2.9x10(7) nucleated cells/kg body weight without any acute or chronic side effects due to rhDNase. On day +35, PCR analysis of bone marrow aspirate showed complete chimerism, and the child engrafted with an absolute neutrophil count greater than 0.5x10(9)/l on day +47. Platelet transfusion independence was achieved on day +120. In conclusion, the supplementation of rhDNase to the washing and resuspension solutions of a thawed UCB unit is effective to prevent cell losses prior to transplantation. However, further investigations must be performed to confirm the safety of this procedure.
The treatment of the myelodysplastic syndrome (MDS) has formerly been supportive, primarily blood transfusions. Treatment with the growth factor erythropoietin, in combination with granulocyte-colony stimulating factor or granulocyte-macrophage colony stimulating factor is accompanied by a decline in the need for blood transfusions in about 40% of the patients. Likewise, the risk of infections is reduced. Immunosuppressive therapy with cyclosporine and antithymocyte globulin is also capable of improving the cytopenia in about 50% of the patients. Allogeneic bone marrow transplantation is the only possibility of cure in MDS, but the procedure is associated with high mortality. This treatment modality is therefore only recommended for younger patients, who are in a complete remission after being treated for acute myeloid leukaemia or are otherwise in a high risk group. The prognosis is poor, with an overall median survival of about 15 months. A simple scoring system has been elaborated and is based upon the percentage of myeloblasts in the bone marrow, a chromosomal analysis, and the number of cell lines with cytopenia. This system is able to distinguish between three distinct groups of patients with a highly different prognosis (low, intermediate, high risk), which is also of importance when evaluating the best treatment for the individual patient.