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INTRODUCTION
Alkaptonuria (AKU) is an inherited metabolic disorder
characterized by the absence of homogentisic acid
oxidase (HGAO) enzyme. The absence of HGAO leads to
the accumulation of homogentisic acid (HGA), produced
during normal metabolism of phenylalanine and tyrosine,
and to an autosomal recessive transmission. The gene for
the enzyme production has been mapped on
chromosome number 3 in the region of bands 3q21-q23.
HGAO – a topologically complex structure – assembles a
functional hexamer arranged as a dimer of trimers. Some
authors have shown the intricate pattern of intra- and
inter-subunit interactions, the extensive surfaces required
for subunit folding and association. Furthermore, they
have shown that this oligomeric enzyme can be
inactivated at multiple levels by single-residue
substitutions (1-4), hence explaining the predominance of
missense mutations (67%) in AKU. Gene mutation
determines the HGA and its polymers are not used and
build up in the body. Some is excreted through the
kidneys (4-8g/day) and causes urine darkening due to the
oxidation of HGA; the rest is deposited in tissues, where it
is toxic. The result is ochronosis, a brown/black
Ochronotic arthropathy with severe articular
involvement necessitates total joint replacement
G. GASPARINI, S. CERCIELLO, M. VASSO, M. CILLO
Department of Orthopaedics and Traumatology Catholic University of Rome, Rome - Italy
ABSTRACT: The aim of this paper is to describe the pathological and clinical features of alkaptonuria
(AKU). AKU clinical presentation is identified as ochronosis, the dark pigmentation of skin and cartilage
that is caused by progressive accumulation of homogentisic acid (HGA) in connective tissues. We also
discuss surgical considerations about joint replacement, which represents the only treatment in the
case of severe articular degeneration.
We report the case of a middle-aged female with both hip and knees affected. She underwent bilateral
hip replacement and right knee arthroplasty. Preoperatively, the patient was examined clinically. Harris
Hip Score (HHS) and Hospital for Special Surgery score (HSS) were completed for both hips, and for
the right knee. Radiographs were made of both hips and the knee, in different projections.
HHS for the left and right sides at the latest follow-up (11 and 9 years after surgery respectively) were
98 and 95. HSS 3.5 years after surgery was 97.
Alkaptonuria is a rare inherited pathology caused by deficiency of the enzyme homogentisic acid
oxidase (HGAO). Skeletal involvement is frequent and causes severe cartilage damage. HGA levels in
blood and urine and articular symptoms make the diagnosis certain. Therapy is still debated: diet offers
good results in young people while high doses of C vitamin seem to reduce the amount of deposits.
Articular localizations usually lead to joint replacement. In this case functional and radiographic results
are excellent even at long follow-up. (Hip International 2006; 16: 305-11)
KEY WORDS: Ochronosis, Homogentisic acid, Joint replacement, Alkaptonuria
Case Report
Hip International / Vol. 16 no. 4, 2006 / pp. 305-311 © Wichtig Editore, 2006