Treatment of Diamond‐Blackfan anaemia with haematopoietic growth factors, granulocyte‐macrophage colony stimulating factor and interleukin 3: sustained remissions following IL‐3

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Abstract
Summary We have treated six transfusion‐dependent, steroid‐unresponsive, Diamond‐Blackfan anaemia (DBA) patients with the recombinant human growth factors granulocyte‐macrophage colony‐stimulating factor (GM‐CSF) and interleukin‐3 (IL‐3), administered sequentially with an interim rest period. GM‐CSF was given at a dose of 500 μg/m2/d subcutaneously for 6 weeks. Three patients increased their absolute reticulocyte counts 1.5‐35‐fold (mean 20.8‐fold) and into the normal range, but only one showed a reduction in transfusion requirements. Between 4 and 25 weeks after discontinuation of GM‐CSF, these six patients were treated with recombinant human IL‐3, at doses of 60 or 125 μg/m2/d subcutaneously for 4‐6 weeks. Three increased their absolute reticulocyte counts from 2‐ to 28‐fold (mean 10.6‐fold) and two required fewer transfusions. One of these two patients has remained transfusion independent for over a year since completion of IL‐3 therapy, and the second patient required infrequent transfusions for 9 months and then became transfusion independent for the subsequent 5 months. The sustained clinical remissions seen in two of the six patients after IL‐3 therapy is very encouraging and further studies in a larger cohort of DBA patients with IL‐3 alone or in combination with GM‐CSF or other growth factors should be carried out.