Shwachman-Diamond syndrome: diarrhea, no longer required?

J Pediatr Hematol Oncol. 2013 Aug;35(6):486-9. doi: 10.1097/MPH.0b013e3182667c13.

Abstract

Exocrine pancreatic insufficiency and diarrhea have been hallmarks in the diagnosis of Shwachman-Diamond syndrome (SDS). We report 2 cases of genetically confirmed SDS in patients who presented with an unusual phenotype. Patient #1 presented with pancytopenia without other system involvement, while patient #2 presented with severe neutropenia, anemia, and a bifid thumb. Neither patient had diarrhea or malabsorption. Both patients had the classic heterozygous mutations c183_184 TA>CT and c.258+2 T>C in the Shwachman-Bodian-Diamond syndrome gene. Incomplete phenotypes may be more common than previously recognized in bone marrow failure syndromes; gastrointestinal symptoms should not be considered a prerequisite for SDS.

Publication types

  • Case Reports

MeSH terms

  • Anemia / genetics
  • Bone Marrow Diseases / complications*
  • Bone Marrow Diseases / genetics
  • Child, Preschool
  • Diarrhea / genetics*
  • Exocrine Pancreatic Insufficiency / complications*
  • Exocrine Pancreatic Insufficiency / genetics
  • Hand Deformities, Congenital / genetics
  • Humans
  • Infant
  • Lipomatosis / complications*
  • Lipomatosis / genetics
  • Male
  • Mutation
  • Pancytopenia / genetics*
  • Phenotype
  • Proteins / genetics
  • Shwachman-Diamond Syndrome
  • Thumb / abnormalities

Substances

  • Proteins
  • SBDS protein, human