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Long-Term Follow-Up and Prognosis of Chronic Granulomatous Disease in Yugoslavia: Is There a Role for Early Bone Marrow Transplantation?

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Abstract

We report the long-term follow-up of 12 pediatric-aged patients with chronic granulomatous disease (CGD). The mean age at the onset of infections was 5 months with a median delay in diagnosis of 2.5 years. Bacille Calmette–Guérin lymphadenitis was the most common presenting infection (6) followed by suppurative lymphadenitis (4), liver abscess (1), or Salmonella sepsis (1). Prophylaxis with cotrimoxazole was recommended to all patients. During the mean follow-up of 10 years (range, 4–23 years) pneumonitis was the most prevalent infection (91%) followed by lymphadenitis (83%), aphtous stomatitis (58%), and liver abscesses (25%). Seven (58%) patients developed chronic lung disease due to grossly delayed diagnosis (3) or poor compliance with antimicrobial prophylaxis (4). Five (41%) patients died during the second decade of life of aspergillosis (3) or chronic lung disease (2). Probability of survival into the third decade of life was estimated to be only 19%. We argue that HLA-identical bone marrow transplantation (BMT), if possible, should be attempted at early age because of significant morbidity and mortality in adolescence. BMT also should be considered in patients who suffer severe infections despite antimicrobial prophylaxis or patients with evidence of chronic lung disease. Possibility of elective BMT from unrelated donors remains to be carefully evaluated.

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Authors and Affiliations

  1. Departments of Pediatric Immunology, Pulmology and Tranfusion, Mother and Child Health Institute, Belgrade, Yugoslavia

    Srdjan Pasic, Aleksandra Minic, Predrag Minic, Dobrila Veljkovic & Bojana Slavkovic

  2. Department of Microbiology and Immunology, The Medical School, University of Newcastle, Newcastle upon-Tyne, United Kingdom

    Desa Lilic

  3. Department of Experimental Immunology, Military Medical Academy, Belgrade, Yugoslavia

    Nada Pejnovic

  4. Department of Paediatrics, Newcastle General Hospital, Newcastle upon-Tyne, England

    Mario Abinun

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  1. Srdjan Pasic
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  2. Aleksandra Minic
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Pasic, S., Minic, A., Minic, P. et al. Long-Term Follow-Up and Prognosis of Chronic Granulomatous Disease in Yugoslavia: Is There a Role for Early Bone Marrow Transplantation?. J Clin Immunol 23, 55–61 (2003). https://doi.org/10.1023/A:1021952315651

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