PMID- 11222364
OWN - NLM
STAT- MEDLINE
DCOM- 20010419
LR  - 20231024
IS  - 0006-4971 (Print)
IS  - 0006-4971 (Linking)
VI  - 97
IP  - 5
DP  - 2001 Mar 1
TI  - Clinical responses to bone marrow transplantation in children with severe 
      osteogenesis imperfecta.
PG  - 1227-31
AB  - Preclinical models have shown that transplantation of marrow mesenchymal cells 
      has the potential to correct inherited disorders of bone, cartilage, and muscle. 
      The report describes clinical responses of the first children to undergo 
      allogeneic bone marrow transplantation (BMT) for severe osteogenesis imperfecta 
      (OI), a genetic disorder characterized by defective type I collagen, osteopenia, 
      bone fragility, severe bony deformities, and growth retardation. Five children 
      with severe OI were enrolled in a study of BMT from human leukocyte antigen 
      (HLA)-compatible sibling donors. Linear growth, bone mineralization, and fracture 
      rate were taken as measures of treatment response. The 3 children with documented 
      donor osteoblast engraftment had a median 7.5-cm increase in body length (range, 
      6.5-8.0 cm) 6 months after transplantation compared with 1.25 cm (range, 1.0-1.5 
      cm) for age-matched control patients. These patients gained 21.0 to 65.3 g total 
      body bone mineral content by 3 months after treatment or 45% to 77% of their 
      baseline values. With extended follow-up, the patients' growth rates either 
      slowed or reached a plateau phase. Bone mineral content continued to increase at 
      a rate similar to that for weight-matched healthy children, even as growth rates 
      declined. These results suggest that BMT from HLA-compatible donors may benefit 
      children with severe OI. Further studies are needed to determine the full 
      potential of this strategy.
FAU - Horwitz, E M
AU  - Horwitz EM
AD  - Cell and Gene Therapy Program, St Jude Children's Research Hospital, Memphis, TN 
      38105, USA. edwin.horwitz@stjude.org
FAU - Prockop, D J
AU  - Prockop DJ
FAU - Gordon, P L
AU  - Gordon PL
FAU - Koo, W W
AU  - Koo WW
FAU - Fitzpatrick, L A
AU  - Fitzpatrick LA
FAU - Neel, M D
AU  - Neel MD
FAU - McCarville, M E
AU  - McCarville ME
FAU - Orchard, P J
AU  - Orchard PJ
FAU - Pyeritz, R E
AU  - Pyeritz RE
FAU - Brenner, M K
AU  - Brenner MK
LA  - eng
GR  - K08 HL 03266/HL/NHLBI NIH HHS/United States
GR  - P30 CA 21765/CA/NCI NIH HHS/United States
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Research Support, U.S. Gov't, P.H.S.
PL  - United States
TA  - Blood
JT  - Blood
JID - 7603509
SB  - IM
MH  - Body Height
MH  - Bone Density
MH  - *Bone Marrow Transplantation/adverse effects/methods
MH  - Female
MH  - Fractures, Spontaneous
MH  - Histocompatibility
MH  - Humans
MH  - Infant
MH  - Male
MH  - Mesoderm/transplantation
MH  - Nuclear Family
MH  - Osteogenesis Imperfecta/complications/physiopathology/*therapy
MH  - Pilot Projects
MH  - Transplantation, Homologous/adverse effects/methods
MH  - Treatment Outcome
EDAT- 2001/02/27 10:00
MHDA- 2001/04/21 10:01
CRDT- 2001/02/27 10:00
PHST- 2001/02/27 10:00 [pubmed]
PHST- 2001/04/21 10:01 [medline]
PHST- 2001/02/27 10:00 [entrez]
AID - S0006-4971(20)56240-X [pii]
AID - 10.1182/blood.v97.5.1227 [doi]
PST - ppublish
SO  - Blood. 2001 Mar 1;97(5):1227-31. doi: 10.1182/blood.v97.5.1227.