Pinsk Maury, Burzynski Jeffrey, Yhap Margaret, Fraser Robert B, Cummings Brian, Ste-Marie Micheline
Department of Pediatrics, University of Virginia Children's Medical Center, Room 2010, MR-4 Building, 300 Lane Road, Charlottesville, VA 22908-0386, USA.
J Pediatr Hematol Oncol. 2002 Dec;24(9):756-8. doi: 10.1097/00043426-200212000-00015.
Glycogen storage disease 1b (GSD 1b) is caused by a deficiency of glucose-6-phosphate translocase and the intracellular accumulation of glycogen. The disease presents with failure to thrive, hepatomegaly, hypoglycemia, lactic acidosis, as well as neutropenia causing increased susceptibility to pyogenic infections. We present a case of a young woman with GSD 1b who developed acute myelogenous leukemia while on long-term granulocyte colony-stimulating factor therapy. The presence of two rare diseases in a single patient raises suspicion that GSD 1b and acute myelogenous leukemia are linked. Surveillance for acute myelogenous leukemia should become part of the long-term follow-up for GSD 1b.
糖原贮积病1b型(GSD 1b)由葡萄糖-6-磷酸转运酶缺乏和糖原在细胞内蓄积所致。该疾病表现为生长发育迟缓、肝肿大、低血糖、乳酸酸中毒,以及中性粒细胞减少导致化脓性感染易感性增加。我们报告一例患有GSD 1b的年轻女性病例,该患者在长期接受粒细胞集落刺激因子治疗期间发生了急性髓系白血病。单一患者同时存在两种罕见疾病引发了对GSD 1b与急性髓系白血病存在关联的怀疑。对急性髓系白血病的监测应成为GSD 1b长期随访的一部分。