title: Successful bone marrow transplantation in a patient with Diamond-Blackfan anemia with co-existing Duchenne muscular dystrophy: a case report.
abstract: BACKGROUND
  Diamond-Blackfan anemia and Duchenne muscular dystrophy are two rare congenital anomalies. Both anomalies occurring in the same child is extremely rare. Allogeneic hematopoietic stem cell transplantation is a well-established therapy for Diamond-Blackfan anemia. However, in patients with Duchenne muscular dystrophy, stem cell therapy still remains experimental.
  
  
  METHODS
  We report the case of a nine-year-old boy of north Indian descent with Diamond-Blackfan anemia and Duchenne muscular dystrophy who underwent successful allogeneic hematopoietic stem cell transplantation. He is transfusion-independent, and his Duchenne muscular dystrophy has shown no clinical deterioration over the past 45 months. His creatine phosphokinase levels have significantly decreased to 300 U/L from 14,000 U/L pre-transplant. The patient is 100% donor chimera in the hematopoietic system, and his muscle tissue has shown 8% to 10.4% cells of donor origin.
  
  
  CONCLUSIONS
  Our patient's Diamond-Blackfan anemia was cured by allogeneic hematopoietic stem cell transplantation. The interesting clinical observation of a possible benefit in Duchenne muscular dystrophy cannot be ruled out. However, further clinical follow-up with serial muscle biopsies and molecular studies are needed to establish this finding.
authors.affiliation: Department of Haematology & Bone Marrow Transplantation, Army Hospital (Research & Referral), Delhi Cantt-110010, India. nairvelu2000@yahoo.com.
authors.forename: Velu
authors.initials: V
authors.lastname: Nair
authors.forename: Satyaranjan
authors.initials: S
authors.lastname: Das
authors.forename: Ajay
authors.initials: A
authors.lastname: Sharma
authors.forename: Sanjeevan
authors.initials: S
authors.lastname: Sharma
authors.forename: Jasmeet
authors.initials: J
authors.lastname: Kaur
authors.forename: Dk
authors.initials: D
authors.lastname: Mishra