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Paroxysmal nocturnal hemoglobinuria in childhood and adolescence-a retrospective analysis of 18 cases

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Abstract

Objective

To assess the clinical and hemotological profile of PNH in children.

Methods

Clinical and laboratory features of children with PNH diagnosed in the past six years at our centre were reviewed. Various investigations done included a complete blood count and peripheral smear examination, plasma hemoglobin, urine hemosiderin, acid ham test, sucrose lysis test, immunophenotyping of erythrocytes by sephadex column gel card and of granulocytes by flow cytometry. There were 18 children with a marked male predominance (M 14: F 4).

Results

Pallor, jaundice, dark urine and bleeding manifestations were the major presenting complaints. One girl suffered an arterial stroke. All children had cytopenia in at least one cell line. Children were treated with danazol, stanazolol, prednisolone and cyclosporin A variously. Overall response rate was 61%. Children with classical PNH performed slightly better with response rates of 66% (6/9) as compared to aplastic anemia-PNH group which has a response rate of 55% (5/9). Amongst various variables only danazol correlated with better response (p=0.029).

Conclusion

PNH is an uncommon disease in children and should be included in the differential diagnosis of children presenting with cytopenia.

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Authors and Affiliations

  1. Department of Hematology, All India Institute of Medical Sciences, New Delhi, India

    Rahul Naithani, Manoranjan Mahapatra, Pankhi Dutta, Rajat Kumar, Hara Prasad Pati & Ved Prakash Choudhry

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  1. Rahul Naithani
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  2. Manoranjan Mahapatra
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  3. Pankhi Dutta
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  4. Rajat Kumar
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  5. Hara Prasad Pati
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  6. Ved Prakash Choudhry
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Correspondence to Manoranjan Mahapatra.

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Naithani, R., Mahapatra, M., Dutta, P. et al. Paroxysmal nocturnal hemoglobinuria in childhood and adolescence-a retrospective analysis of 18 cases. Indian J Pediatr 75, 575–578 (2008). https://doi.org/10.1007/s12098-008-0111-9

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  • DOI: https://doi.org/10.1007/s12098-008-0111-9

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