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

Naithani, Rahul; Mahapatra, Manoranjan; Dutta, P. K.; Kumar, Rajat; Pati, Hara Prasad; Choudhry, V. P.

doi:10.1007/s12098-008-0111-9

Cited by 20 publications

(34 citation statements)

References 23 publications

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“…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] .…”

Section: Discussionsupporting

confidence: 92%

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

Shi

et al. 2015

Acta Haematol

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Objective: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease, especially in children. To characterize the clinical presentations and survival, we performed a retrospective analysis of pediatric patients. Methods: We reviewed 55 pediatric patients with PNH referred to our hospital from January 1990 through June 2012 to assess clinical presentations, survival, and differences among subcategories. Results: The overall survival 10 years after diagnosis estimated via the Kaplan-Meier method was 77.6%. The cohort of patients was divided into subcategories of classic PNH, PNH/aplastic anemia (AA), and subclinical PNH (PNH-sc)/AA based on the recently proposed PNH working clinical classification. We found that patients with classic PNH and PNH/AA had larger PNH clones and many more parameters of hemolysis, but patients with PNH-sc/AA had smaller PNH clones, fewer parameters of hemolysis, and a higher rate of bone marrow failure. Our results revealed a high rate of bone marrow failure and a low rate of hemoglobinuria at presentation. Furthermore, thrombotic events were not observed in our patients, which is significantly different from the rate seen in Caucasian patients. Additionally, pediatric patients with PNH may develop bone marrow cytogenetic abnormalities. Conclusion: This study provides insight into Chinese pediatric PNH patients and may aid in setting up individualized therapeutic regimens.

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Section: Discussionsupporting

confidence: 92%

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

Shi

et al. 2015

Acta Haematol

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“…PNH is a rare acquired disorder of hematopoietic stem cells, which mainly presents as a disease of adults and, to a less extent, of childhood and adolescence [3,[21][22][23]. Our report showed that the median age of PNH patients at diagnosis was 32 years, which was similar to some reports [2,7], but younger than others [1,6].…”

Section: Discussionsupporting

confidence: 87%

“…Thrombotic complications in PNH patients can arise in hepatic, cerebral, and deep limb veins [1,24,25]. The development of thrombosis was common in Caucasian but rare in Asian patients with PNH [8][9][10]21]. Our study documented crude incidence of thrombotic events as 3.5%, which was significantly lower than previous reports [2,7], but still remained to be a grave prognostic feature of PNH.…”

Section: Discussionmentioning

confidence: 45%

Clinical features and prognostic factors of Asian patients with paroxysmal nocturnal hemoglobinuria: results from a single center in China

Shi

et al. 2012

Ann Hematol

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Although all patients with paroxysmal nocturnal hemoglobinuria (PNH) have acquired mutations in the phosphatidylinositol glycan class-A(PIG-A)gene, their clinical courses are highly variable. We reviewed 280 PNH cases referred to our hospital from January 1990 through June 2010 to assess clinical presentations, prognostic factors influencing survival, difference among subcategories, and clinical significance of PNH clone size. The overall survival at 10 years after diagnosis estimated by Kaplan-Meier was 77.6%. Both univariate and multivariate analyses identified risk factors affecting survival, including age >40 years, absolute neutrophil count<0.5 × 10(9) cells/L, development of thrombotic events, evolution to myelodysplastic syndrome or acute myelogenous leukemia, and recurrent infections. The cohort of patients were divided into subcategories of classic PNH, PNH/AA, and PNH-sc/AA based on the recent proposed PNH working clinical classification, hemoglobinuria as the initial symptomatic manifestation was high up to 82.0% in classic PNH subcategory, whereas only as low as 1.4% in PNH-sc/AA subcategory; the frequencies of infectious (26.0%) and bleeding symptoms (14.0%) in classic PNH subcategory were significantly less than those in PNH/AA (25.3% and 51.7%, respectively) and PNH-sc/AA (48.3% and 83.2%, respectively) subcategories. Our results revealed that large PNH clone was associated with increased risks for hemoglobinuria and thrombosis, whereas small PNH clone was associated with bone marrow failure. Thus, this study may shed insights into Chinese PNH patients to set up individually therapeutic regimens.

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“…1,[5][6][7]15,16 Our data confirm that thrombosis (TE and MAVE) is an important complication of PNH not only in adults (2.26 per 100 person-years), but also in children (1.23 per 100 personyears). However, myelodysplastic syndrome or myelodysplastic features previously reported in two smaller series 5,7 were not confirmed in our registry cohort.…”

Section: Letters To the Editorsupporting

confidence: 76%

Different clinical characteristics of paroxysmal nocturnal hemoglobinuria in pediatric and adult patients

et al. 2016

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

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

Cited by 20 publications

References 23 publications

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

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

Clinical features and prognostic factors of Asian patients with paroxysmal nocturnal hemoglobinuria: results from a single center in China

Different clinical characteristics of paroxysmal nocturnal hemoglobinuria in pediatric and adult patients

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