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Related Experiment Videos

Bone marrow transplants in genetic diseases.

J R Hobbs1

  • 1Westminster Children's Bone Marrow Team, Department of Immunology, Charing Cross and Westminster Medical School, London, United Kingdom.

European Journal of Pediatrics
|January 1, 1992
PubMed
Summary

Bone marrow transplantation (BMT) offers a lifelong solution for over 50 genetic diseases, with elective procedures achieving 95% success. Early diagnosis and evaluation for BMT are crucial for pediatric patients.

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Area of Science:

  • Hematology
  • Genetics
  • Pediatrics

Background:

  • Bone marrow transplantation (BMT) was first advocated in 1978 for genetic metabolic diseases.
  • Early BMT efforts evolved from clinical experience, highlighting the need for matched sibling donors.
  • The concept of BMT involves establishing a permanent donor marrow 'factory' for lifelong correction.

Purpose of the Study:

  • To outline the principles and applications of displacement bone marrow transplantation (DBMT).
  • To review the success and limitations of DBMT in treating genetic disorders.
  • To provide guidelines for future extensions of BMT, including gene therapy.

Main Methods:

  • Review of historical data and clinical experience with DBMT.
  • Analysis of treatment outcomes for patients with various genetic diseases.
  • Assessment of the applicability of DBMT to inborn errors.

Main Results:

  • DBMT has successfully treated over 700 patients with approximately 50 disabling diseases worldwide.
  • DBMT is applicable to about 7% of known inborn errors.
  • Elective DBMT demonstrates superior success rates of about 95%.

Conclusions:

  • DBMT provides a potentially curative, lifelong treatment for selected genetic metabolic diseases.
  • The success of DBMT encourages earlier diagnosis and transplant evaluation in pediatric patients.
  • Future directions include expanding BMT applications and exploring gene transplants.

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