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

Ge, Meili; Shi, Jun; Li, Xingxin; Shao, Yijun; Huang, Jinbo; Huang, Zhendong; Nie, Neng; Zheng, Yizhou

doi:10.1159/000369773

Cited by 5 publications

(3 citation statements)

References 24 publications

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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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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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“…14 The true incidence of TE in pediatric PNH has been difficult to determine; previous pediatric series have reported values as low as 0-4% in Asian cohorts and as high as 50% among Caucasians. 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

Urbano-Ispizúa

Muus

Schrezenmeier

et al. 2015

Blood

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Introduction: Studies of children with paroxysmal nocturnal hemoglobinuria (PNH) are scarce and include a very limited number of patients. The objective of this analysis was to describe characteristics of PNH at enrollment for the largest available registry of pediatric patients, and to compare demographic and clinical characteristics with those of adult patients. Methods: The International PNH Registry is a prospective, multi-center worldwide, observational study of patients with a PNH clone of 0.01-100%. Data are collected from patient medical records at the time of Registry enrollment and every six months thereafter. Adult patients were ³18 years of age at enrollment and disease start and pediatric patients were <18 years at enrollment. Demographics and clinical parameters in patients untreated with eculizumab at enrollment for the two age cohorts were compared using the Wilcoxon-Mann-Whitney test for medians and PearsonÕs chi-square for frequencies. The rate of thrombotic events (TE) between disease start (defined as the earliest reported PNH symptoms, granulocyte clone, or PNH diagnosis) and enrollment was calculated per 100 person-years. Results: As of March 2, 2015, a total of 2,184 patients were eligible for analysis: 94 (4.3%) pediatric patients and 2,090 (95.7%) adult patients. Median age (range) at enrollment was 14.0 years (3-17) in pediatrics and 45.5 years (18-100) in adults; median disease duration was 0.7 years and 2.1 years, respectively (p<0.001). More pediatric than adult patients had a PNH clone of <10% and severe cytopenia (Table). Pediatric patients had lower percent of reticulocytes compared with adults (2.1% vs. 2.6%, respectively; p=0.015). History of aplastic or hypoplastic anemia was more frequent in pediatric than adult patients (76.5% vs 54.4%, respectively; p<0.001). History of TE and any major adverse vascular event was less frequent in pediatrics (2.1% vs 8.7%; p=0.025, and 4.3% vs. 14.4%; p=0.005). The rate of TE between disease start and enrollment was lower in pediatric patients, but not statistically significant: 1.4 per 100 person-years (95%CI 0.2-5.2) compared to adult patients (2.3 per 100 person-years (95%CI 2.0-2.6). More pediatric patients than adults had abdominal pain at enrollment. Conclusions: The International PNH Registry provides the largest available pediatric cohort of patients with a PNH clone to characterize this understudied population and demonstrate an important disease burden. Pediatric patients were more likely to have smaller PNH clones and a higher component of aplastic/hypoplastic anemia. Pediatric patients had fewer vascular events. These findings may reflect the natural evolution of the disease and can be useful in the clinical management of PNH. Table 1. Clinical Characteristics at Enrollment of Pediatric and Adult Patients with PNH Pediatric(n=94) Adult(n=2,090) P-value Clone size (percent GPI-deficient granulocytes), n (%)<10% 10 to < 50% ³50% 47 (55.3) 16 (18.8) 22 (25.9) 550 (38.3) 322 (22.4) 565 (39.3) 0.006* Cytopenia status, n (%)None (neutrophils ³ 1.5 x 109/L and platelets ³100 x 109/L) Moderate (neutrophils <1.5 x 109/L or platelets <100 x 109/L) Severe (neutrophils <0.5 x 109/L or platelets <20x109/L) 22 (29.3) 28 (37.3) 25 (33.3) 735 (42.2) 784 (45.1) 221 (12.7) <0.001* Percent reticulocytesMedian (Q1, Q3) 2.1 (1.1, 3.5) 2.6 (1.6, 4.6) 0.015 Hemolytic status, n (%)Hemolytic (LDH ³1.5 x ULN and/or reticulocytes ³60 x 109/L) Not hemolytic (LDH <1.5 x ULN and reticulocytes <60 x 109/L) 33 (58.9) 23 (41.1) 1,038 (65.3) 551 (34.7) NS LDH Ratio, n (%)<1.5 x ULN³1.5 x ULN 30 (58.8) 21 (41.2) 684 (47.0) 770 (53.0) NS History of TE, n (%)Yes No 2 (2.1) 92 (97.9) 181 (8.7) 1,902 (91.3) 0.025 Rate of TENumber of TE, n Person-years (disease start to enrollment) Rate/100 person-years (95% CI) 2 139.9 1.4 (0.2-5.2) 255 11,119.8 2.3 (2.0-2.6) NS History of MAVE, n (%) 4 (4.3) 300 (14.4) 0.005 GPI, glycosylphosphatidylinositol; LDH, lactate dehydrogenase; MAVE, major adverse vascular event; TE, thrombotic event; ULN, upper limit of normal *P-values for clone size and cytopenia status represent overall comparison of categories. Disclosures Muus: Alexion Pharmaceuticals: Honoraria. Schrezenmeier:Alexion Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau. Almeida:Celgene: Consultancy; Novartis: Consultancy; Bristol Meyer Squibb: Speakers Bureau; Shire: Speakers Bureau. Wilson:Alexion Pharmaceuticals: Employment. Ware:Bayer Pharmaceuticals: Consultancy; Biomedomics: Research Funding; Eli Lilly: Other: DSMB membership; Bristol Myers Squibb: Research Funding.

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“…2,10,13 Asian children with PNH appear to be less at risk. 16 Anemia is often associated with symptoms including weakness, paleness, shortness of breath, and fatigue. In our cohort of 150 children presenting with thrombosis, 24.5% of the children had an anemia and 15% indicated weakness or fatigue (►Fig.…”

Section: Discussionmentioning

confidence: 99%

Paroxysmal Nocturnal Hemoglobinuria: An Underestimated Cause of Pediatric Thromboembolism

Griesser

Myskiw

Streif

2020

TH Open

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Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic disease caused by complement-mediated hemolysis. Clinical symptoms include intravascular hemolysis, nocturnal hemoglobinuria, thromboses, cytopenia, fatigue, abdominal pain, and a strong tendency toward bone marrow failure. It is a rare disease, especially in children, with high mortality rates without appropriate treatment.We here present the case of a 17-year-old girl with unprovoked muscle vein thrombosis. Flow cytometric analysis showed deficiency of glycosyl-phosphatidylinositol-anchored membrane proteins on all three hematopoietic cell lines and confirmed the diagnosis of PNH. Treatment with the monoclonal antibody eculizumab achieved long-term remission.As flow cytometry is normally not part of the routine diagnostics for pediatric thrombosis, awareness is crucial and PNH is important to consider in all children with thrombosis at atypical sites and abnormalities in blood counts with regard to hemolysis and cytopenia.

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

Cited by 5 publications

References 24 publications

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

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

Different Clinical Characteristics of Paroxysmal Nocturnal Hemoglobinuria in Pediatric and Adult Patients

Paroxysmal Nocturnal Hemoglobinuria: An Underestimated Cause of Pediatric Thromboembolism

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