Clinicopathological profile of paroxysmal nocturnal haemoglobinuria clone‐positive aplastic anaemia paediatric patients—A single centre study from North India

Rahman, Khaliqur; Mittal, Navkirti; Gupta, Ritu; Kumar, Sandeep; Gupta, Tanvi; Gupta, Anshul; Nityanand, Soniya

doi:10.1111/ijlh.12875

Cited by 2 publications

(9 citation statements)

References 17 publications

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“…In addition, sequential omission of individual studies was conducted as a sensitivity analysis to test the heterogeneity. Notably, the response rate in the PNH+ group was higher than that in the PNH– group (OR 3.31; 95% CI 2.48–4.41; p <0.00001) and heterogeneity decreased most ( I 2 = 19%) when the study by Rahman et al [17] was excluded from the analysis (Table 4). The characteristics provided were not significantly different from those of the other studies.…”

Section: Resultsmentioning

confidence: 99%

“…Due to insufficient individual data and limited sample sizes, our statistical analysis was restricted to the “children” and “definition of PNH clones >1%” subsets, which showed no significant differences and did not decrease heterogeneity; in this regard, we failed to explain the heterogeneity by age or different definitions of PNH clones. Sensitivity analysis demonstrated that the study by Rahman et al [17] was the most responsible for the heterogeneity and that the survival or loss of follow-up bias may have originated from this heterogeneity.…”

Section: Discussionmentioning

confidence: 99%

“…The initial search identified 1,452 references from the databases, 55 records from Clinicaltrials.gov, and 132 abstracts from the ASH/EBMT (European Society for Blood and Marrow Transplantation) annual meetings. After duplicates were excluded and titles or abstracts were screened, the full text was critically appraised for 54 references, and 11 cohort studies were ultimately chosen for this meta-analysis [15][16][17][25][26][27][28][29][30][31][32]. The selection process is illustrated in Figure 1.…”

Section: Data Searchingmentioning

confidence: 99%

“…With regard to PNH clones, they have been found to be predictive of response in some but not all studies [3]. Furthermore, several studies have reported contradictory results regarding the impact of PNH clones on the IST response [15][16][17]. Therefore, it is important to ensure the predictive value of PNH clones for AA with IST with a systematic review and meta-analysis.…”

Section: Introductionmentioning

confidence: 99%

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PNH Clones for Aplastic Anemia with Immunosuppressive Therapy: A Systematic Review and Meta-Analysis

Pan

et al. 2020

Acta Haematol

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Objectives: PNH clones, also aptly called “escape clones,” are evidence of acquired immune-mediated bone marrow failure and have a high prevalence in patients with aplastic anemia (AA). Several studies have reported contradictory results regarding the impact of PNH clones on AA patients with immunosuppression treatment, and PNH clones have not been confirmed as positive predictors of response in the AA guidelines of the British Society for Standards in Haematology. Methods: We performed a meta-analysis to address this issue by searching for articles in PubMed, EMBASE, The Cochrane Library, Web of Science, and ClinicalTrials.gov, and for abstracts from the annual meetings of the American Society of Hematology and the European Hematology Association. We included 1,236 participants from 11 cohort-controlled studies. Our primary outcome was the 6-month hematologic response with a secondary outcome of the mortality rate within 3 months. Results: A better response rate was observed in the PNH+ group than in the PNH– group (odds ratio [OR] 2.85; 95% confidence interval [CI] 2.17–3.75; p < 0.00001), and further subgroup analysis strengthened the outcome, with minor heterogeneity in non-Asian countries. In contrast, the early mortality was not significantly different between the PNH+ and PNH– groups (OR 0.54; 95% CI 0.26–1.10; p = 0.09). Conclusions: The meta-analysis suggested an evidence-based role for PNH clones in predicting a better response in AA patients with immunosuppression.

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Section: Resultsmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Data Searchingmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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PNH Clones for Aplastic Anemia with Immunosuppressive Therapy: A Systematic Review and Meta-Analysis

Pan

et al. 2020

Acta Haematol

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“…Paroxysmal nocturnal haemoglobinuria (PNH) is an acquired haematopoietic stem cell disorder. 1 It is rare in children and results from a somatic mutation in the glycophosphatidylinositol (GPI) glycan A gene on the short arm of the X chromosome. 1 , 2 , 3 The GPI glycan A gene encodes GPI-anchored markers, including CD55 and CD59, which protect cells from complement-mediated attack.…”

Section: Introductionmentioning

confidence: 99%

Childhood aplastic anaemia with paroxysmal nocturnal haemoglobinuria clones: A retrospective single-centre study in South Africa

et al. 2022

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Background: Paroxysmal nocturnal haemoglobinuria (PNH) clones in children are rare but commonly associated with aplastic anaemia (AA) and myelodysplasia.Objective: This study aimed to determine the prevalence of PNH clones in paediatric patients with idiopathic AA, identify differences in clinical and laboratory features and outcomes, and determine the impact of clone size on clinical presentation.Methods: Patients with confirmed idiopathic AA who were tested for PNH between September 2013 and January 2018 at the Inkosi Albert Luthuli Central Hospital, Durban, KwaZulu-Natal, South Africa, were included. PNH clones were detected in neutrophils and monocytes by flow cytometry using fluorescent aerolysin, CD24, CD66b and CD14.Results: Twenty-nine children with AA were identified and 11 were excluded. Ten patients (10/18, 55.6%) had PNH clones ranging from 0.11% to 24%. Compared to the PNH-negative group, these children were older (median: 10 years vs 4 years, p = 0.02) and had significantly lower total white cell counts (median 1.7 × 109/L vs 3.2 × 109/L; p = 0.04). There was no difference in median absolute neutrophil count or haemoglobin concentration. Four patients in each group received immunosuppressive therapy (IST). At six months, all four patients with PNH clones had responded, compared to one in the PNH-negative group.Conclusion: More than half of children with AA had a PNH clone. The size of the clone did not impact clinical severity; however, IST use may positively impact prognosis. We recommend early initiation of IST in patients with AA to avoid delays associated with human leukocyte antigen typing.

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Clinicopathological profile of paroxysmal nocturnal haemoglobinuria clone‐positive aplastic anaemia paediatric patients—A single centre study from North India

Abstract: There is a high prevalence of PNH clones in paediatric AA patients, which in a majority of cases are of small clone sizes. The use of immunosuppressive therapy does not show a better outcome as compared to PNH-negative cases.

Cited by 2 publications

References 17 publications

PNH Clones for Aplastic Anemia with Immunosuppressive Therapy: A Systematic Review and Meta-Analysis

PNH Clones for Aplastic Anemia with Immunosuppressive Therapy: A Systematic Review and Meta-Analysis

Childhood aplastic anaemia with paroxysmal nocturnal haemoglobinuria clones: A retrospective single-centre study in South Africa

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