Stem cell transplantation for paroxysmal nocturnal haemoglobinuria in childhood

Abstract

Summary. Paroxysmal nocturnal haemoglobinuria (PNH) is a clonal haematopoietic disorder characterized by chronic or intermittent intravascular haemolysis, variable cytopenia and an increased risk of thrombosis. Stem cell transplantation (SCT) is a curative therapeutic option, but its risks must be carefully weighed against the natural course of PNH. World-wide experience with SCT for PNH in the paediatric age group is scarce. We report on two adolescents suffering from PNH with life-threatening complications who were successfully transplanted from unrelated donors. Indications and techniques of SCT in childhood PNH are discussed and an overview of the literature is given.

Clinical Features and Survival of Asian Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria: Results from a Single Center in China

“…Both sexes can be affected, though our series revealed a slight male preponderance of PNH. Though usually described as hemolytic anemia, PNH-associated cytopenias were common as 85.5% of patients presented thrombocytopenia, neutropenia, or both in our study, which is similar to the results of previous reports [13,17,18,21,22] .…”

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

“…We could retrieve only 52 cases from Pubmed (Table 3). 3,[5][6][7][8][9][10][11][12][13][14][15] There is a dramatic male predominance in the present study cohort. This however may be biased as male children are more often brought to medical attention in developing countries.…”

Outcomes of paroxysmal nocturnal hemoglobinuria in the pediatric age group in a resource‐constrained setting

“…PNH is a rare disease especially in children . AA or MDS associated with PNH has been reported in about 24‐33% of adults …”