Description

Lucarelli et al evaluated outcomes of patients with thalassemia who underwent bone marrow transplantation. They identified factors impacting the outcome which help to prioritize patients for the procedure. The authors are from Ospedale di Pesaro in Italy.


 

Patient population: homozygous beta-thalassemia

 

Factors associated with a poor outcome:

(1) portal fibrosis

(2) one or both of the following:

(2a) hepatomegaly (> 2 cm, presumably below the right costal margin)

(2b) inadequate chelation therapy

 

Number of Factors Present

3 Year Survival

0

94%

1

80%

2

61%

 

 

Additional factors associated with a better outcome:

(1) age < 16 years of age

(2) HLA-identical donor

 

Limitations:

• Survival following bone marrow transplantation probably has improved since the paper was written.

 


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