Haematological abnormalities in Shwachman-Diamond syndrome

Br J Haematol. 1996 Aug;94(2):279-84. doi: 10.1046/j.1365-2141.1996.d01-1788.x.

Abstract

We have analysed the haematological parameters in 21 patients with Shwachman-Diamond syndrome (SDS) seen over a 25-year period at our institution. Neutropenia, although present in all patients, was intermittent in two-thirds, constant in the rest and was associated with impaired chemotaxis in all of those patients tested. Fetal haemoglobin (HbF) was elevated in 80% of the patients at some stage, and anaemia and thrombocytopenia was documented in 66% and 24% respectively. Bone marrow samples were taken in over half of the patients. Myelodysplastic syndrome (MDS) developed in seven (33%) patients, five of whom had acquired clonal structural chromosome abnormalities in their bone marrows. In five of the patients with MDS (24%) transformation to acute myeloid leukaemia occurred. Like other constitutional bone marrow failure syndromes. SDS has a predilection to leukaemic transformation hitherto assumed to be in the region of 5-10%. The data presented here suggest that this figure probably represents an underestimate. Shwachman-Diamond syndrome is an interesting model of leukaemia development and greater understanding of the clinical spectrum of this rare disorder should produce further insights into its pathobiology.

MeSH terms

  • Adolescent
  • Adult
  • Bone Marrow Diseases / blood
  • Bone Marrow Diseases / complications*
  • Bone Marrow Diseases / genetics
  • Child, Preschool
  • Chromosome Aberrations
  • Exocrine Pancreatic Insufficiency / blood
  • Exocrine Pancreatic Insufficiency / complications*
  • Exocrine Pancreatic Insufficiency / genetics
  • Female
  • Fetal Hemoglobin / analysis
  • Growth Disorders / blood
  • Growth Disorders / complications*
  • Growth Disorders / genetics
  • Hematologic Diseases / etiology*
  • Humans
  • Infant
  • Leukemia, Myeloid / etiology
  • Male
  • Myelodysplastic Syndromes / etiology
  • Prognosis
  • Syndrome

Substances

  • Fetal Hemoglobin