PROSPECTIVE MULTICENTRIC EVALUATION OF THE CURRENT MEDICAL INDICATION FOR PAROXYSMAL NOCTURAL HEMOGLOBINURIA DIAGNOSTIC SCREENING
(Abstract release date: 05/19/16)
EHA Library. Orfao A. 06/09/16; 132556; E1007
Prof. Dr. Alberto Orfao
Contributions
Contributions
Abstract
Abstract: E1007
Type: Eposter Presentation
Background
Although consensus guidelines have been proposed in 2010 for the diagnostic screening of paroxysmal nocturnal hemoglobinuria (PNH) by flow cytometry (FCM), so far no study has investigated the efficiency of such medical indications in multicentric vs. reference laboratory settings.
Aims
To evaluated the efficiency of the current consensus medical indications for diagnostic screening of PNH by FCM
Methods
Overall, information about 3,938 samples from an identical number of individuals prospectively submitted between January 2011 and December 2014 for diagnostic screening of PNH by flow cytometry was collected at 24 flow cytometry laboratories in Spain which participate in the PNH-External Quality Assurance Program of the Iberian Society of Cytometry (1,718 samples) plus one reference laboratory in Sao Paulo, Brazil (2,220 samples). The following GPI-associated markers were analyzed: FLAER (analyzed in 87% of the cases), CD14 (98%), CD16 (37%), CD24 (93%), and/or CD157 (5%). In those cases with GPI-deficient mature neutrophils and monocytes, expression of CD59 (100% of cases) was also analyzed on red blood cells.
Results
Overall, diagnostic screening based on consensus medical indications was highly efficient (567 PNH+/3,938 screened cases; 14% PNH+ samples) both in the multicenter setting in Spain (10%) and reference laboratory in Brazil (16%). Although GPI-deficient cells were found within all age groups, a significantly higher frequency of PNH+ cases was observed among the screened PB samples from individuals ≤40 years vs. older cases (18% vs. 11%; p<0.001) Estimated annual incidence of new PNH cases was be of ≈2.5 cases/million individuals per year. When patients with previously diagnosed hematological associated disorders (mostly AA and MDS) were excluded from the analysis, the annual incidence of PNH was of 0.6 cases per million individuals per year.The highest frequency of PNH+ cases was observed among patients screened because of bone marrow (BM) failure syndrome (33%), particularly among those with mainly aplastic anemia (243/541; 45%) and to a less extent also myelodysplastic syndrome (26/266; 10%). Among the other individuals studied, the most efficient medical indications for PNH screening included: hemoglobinuria (35/73; 48%), unexplained cytopenias including anemia (88/393; 22%), non-immune hemolytic anemia (71/382; 19%), and thrombosis associated to (non-hemolytic) anemia and/or another cytopenia (10/73; 14%). PNH+ cases was less commonly observed among patients with chronic myeloproliferative neoplasms (1/21; 4.8%), unexplained cytopenias in the absence of anemia (39/772; 5.1%) or unspecified anemia (17/468; 3.6%). In contrast, only a minor fraction of the patients who had been submitted for PNH testing because of unexplained thrombosis in the absence of cytopenia were positive (3/800; 0.4%).
Conclusion
In summary, our results demonstrate that the current medical indications for PNH screening by FCM are highly efficient, although improved screening algorithms are needed for patients presenting with thrombosis and normal blood cell counts.
Session topic: E-poster
Keyword(s): Diagnosis, Medical patients, Paroxysmal nocturnal hemoglobinuria (PNH)
Type: Eposter Presentation
Background
Although consensus guidelines have been proposed in 2010 for the diagnostic screening of paroxysmal nocturnal hemoglobinuria (PNH) by flow cytometry (FCM), so far no study has investigated the efficiency of such medical indications in multicentric vs. reference laboratory settings.
Aims
To evaluated the efficiency of the current consensus medical indications for diagnostic screening of PNH by FCM
Methods
Overall, information about 3,938 samples from an identical number of individuals prospectively submitted between January 2011 and December 2014 for diagnostic screening of PNH by flow cytometry was collected at 24 flow cytometry laboratories in Spain which participate in the PNH-External Quality Assurance Program of the Iberian Society of Cytometry (1,718 samples) plus one reference laboratory in Sao Paulo, Brazil (2,220 samples). The following GPI-associated markers were analyzed: FLAER (analyzed in 87% of the cases), CD14 (98%), CD16 (37%), CD24 (93%), and/or CD157 (5%). In those cases with GPI-deficient mature neutrophils and monocytes, expression of CD59 (100% of cases) was also analyzed on red blood cells.
Results
Overall, diagnostic screening based on consensus medical indications was highly efficient (567 PNH+/3,938 screened cases; 14% PNH+ samples) both in the multicenter setting in Spain (10%) and reference laboratory in Brazil (16%). Although GPI-deficient cells were found within all age groups, a significantly higher frequency of PNH+ cases was observed among the screened PB samples from individuals ≤40 years vs. older cases (18% vs. 11%; p<0.001) Estimated annual incidence of new PNH cases was be of ≈2.5 cases/million individuals per year. When patients with previously diagnosed hematological associated disorders (mostly AA and MDS) were excluded from the analysis, the annual incidence of PNH was of 0.6 cases per million individuals per year.The highest frequency of PNH+ cases was observed among patients screened because of bone marrow (BM) failure syndrome (33%), particularly among those with mainly aplastic anemia (243/541; 45%) and to a less extent also myelodysplastic syndrome (26/266; 10%). Among the other individuals studied, the most efficient medical indications for PNH screening included: hemoglobinuria (35/73; 48%), unexplained cytopenias including anemia (88/393; 22%), non-immune hemolytic anemia (71/382; 19%), and thrombosis associated to (non-hemolytic) anemia and/or another cytopenia (10/73; 14%). PNH+ cases was less commonly observed among patients with chronic myeloproliferative neoplasms (1/21; 4.8%), unexplained cytopenias in the absence of anemia (39/772; 5.1%) or unspecified anemia (17/468; 3.6%). In contrast, only a minor fraction of the patients who had been submitted for PNH testing because of unexplained thrombosis in the absence of cytopenia were positive (3/800; 0.4%).
Conclusion
In summary, our results demonstrate that the current medical indications for PNH screening by FCM are highly efficient, although improved screening algorithms are needed for patients presenting with thrombosis and normal blood cell counts.
Session topic: E-poster
Keyword(s): Diagnosis, Medical patients, Paroxysmal nocturnal hemoglobinuria (PNH)
Abstract: E1007
Type: Eposter Presentation
Background
Although consensus guidelines have been proposed in 2010 for the diagnostic screening of paroxysmal nocturnal hemoglobinuria (PNH) by flow cytometry (FCM), so far no study has investigated the efficiency of such medical indications in multicentric vs. reference laboratory settings.
Aims
To evaluated the efficiency of the current consensus medical indications for diagnostic screening of PNH by FCM
Methods
Overall, information about 3,938 samples from an identical number of individuals prospectively submitted between January 2011 and December 2014 for diagnostic screening of PNH by flow cytometry was collected at 24 flow cytometry laboratories in Spain which participate in the PNH-External Quality Assurance Program of the Iberian Society of Cytometry (1,718 samples) plus one reference laboratory in Sao Paulo, Brazil (2,220 samples). The following GPI-associated markers were analyzed: FLAER (analyzed in 87% of the cases), CD14 (98%), CD16 (37%), CD24 (93%), and/or CD157 (5%). In those cases with GPI-deficient mature neutrophils and monocytes, expression of CD59 (100% of cases) was also analyzed on red blood cells.
Results
Overall, diagnostic screening based on consensus medical indications was highly efficient (567 PNH+/3,938 screened cases; 14% PNH+ samples) both in the multicenter setting in Spain (10%) and reference laboratory in Brazil (16%). Although GPI-deficient cells were found within all age groups, a significantly higher frequency of PNH+ cases was observed among the screened PB samples from individuals ≤40 years vs. older cases (18% vs. 11%; p<0.001) Estimated annual incidence of new PNH cases was be of ≈2.5 cases/million individuals per year. When patients with previously diagnosed hematological associated disorders (mostly AA and MDS) were excluded from the analysis, the annual incidence of PNH was of 0.6 cases per million individuals per year.The highest frequency of PNH+ cases was observed among patients screened because of bone marrow (BM) failure syndrome (33%), particularly among those with mainly aplastic anemia (243/541; 45%) and to a less extent also myelodysplastic syndrome (26/266; 10%). Among the other individuals studied, the most efficient medical indications for PNH screening included: hemoglobinuria (35/73; 48%), unexplained cytopenias including anemia (88/393; 22%), non-immune hemolytic anemia (71/382; 19%), and thrombosis associated to (non-hemolytic) anemia and/or another cytopenia (10/73; 14%). PNH+ cases was less commonly observed among patients with chronic myeloproliferative neoplasms (1/21; 4.8%), unexplained cytopenias in the absence of anemia (39/772; 5.1%) or unspecified anemia (17/468; 3.6%). In contrast, only a minor fraction of the patients who had been submitted for PNH testing because of unexplained thrombosis in the absence of cytopenia were positive (3/800; 0.4%).
Conclusion
In summary, our results demonstrate that the current medical indications for PNH screening by FCM are highly efficient, although improved screening algorithms are needed for patients presenting with thrombosis and normal blood cell counts.
Session topic: E-poster
Keyword(s): Diagnosis, Medical patients, Paroxysmal nocturnal hemoglobinuria (PNH)
Type: Eposter Presentation
Background
Although consensus guidelines have been proposed in 2010 for the diagnostic screening of paroxysmal nocturnal hemoglobinuria (PNH) by flow cytometry (FCM), so far no study has investigated the efficiency of such medical indications in multicentric vs. reference laboratory settings.
Aims
To evaluated the efficiency of the current consensus medical indications for diagnostic screening of PNH by FCM
Methods
Overall, information about 3,938 samples from an identical number of individuals prospectively submitted between January 2011 and December 2014 for diagnostic screening of PNH by flow cytometry was collected at 24 flow cytometry laboratories in Spain which participate in the PNH-External Quality Assurance Program of the Iberian Society of Cytometry (1,718 samples) plus one reference laboratory in Sao Paulo, Brazil (2,220 samples). The following GPI-associated markers were analyzed: FLAER (analyzed in 87% of the cases), CD14 (98%), CD16 (37%), CD24 (93%), and/or CD157 (5%). In those cases with GPI-deficient mature neutrophils and monocytes, expression of CD59 (100% of cases) was also analyzed on red blood cells.
Results
Overall, diagnostic screening based on consensus medical indications was highly efficient (567 PNH+/3,938 screened cases; 14% PNH+ samples) both in the multicenter setting in Spain (10%) and reference laboratory in Brazil (16%). Although GPI-deficient cells were found within all age groups, a significantly higher frequency of PNH+ cases was observed among the screened PB samples from individuals ≤40 years vs. older cases (18% vs. 11%; p<0.001) Estimated annual incidence of new PNH cases was be of ≈2.5 cases/million individuals per year. When patients with previously diagnosed hematological associated disorders (mostly AA and MDS) were excluded from the analysis, the annual incidence of PNH was of 0.6 cases per million individuals per year.The highest frequency of PNH+ cases was observed among patients screened because of bone marrow (BM) failure syndrome (33%), particularly among those with mainly aplastic anemia (243/541; 45%) and to a less extent also myelodysplastic syndrome (26/266; 10%). Among the other individuals studied, the most efficient medical indications for PNH screening included: hemoglobinuria (35/73; 48%), unexplained cytopenias including anemia (88/393; 22%), non-immune hemolytic anemia (71/382; 19%), and thrombosis associated to (non-hemolytic) anemia and/or another cytopenia (10/73; 14%). PNH+ cases was less commonly observed among patients with chronic myeloproliferative neoplasms (1/21; 4.8%), unexplained cytopenias in the absence of anemia (39/772; 5.1%) or unspecified anemia (17/468; 3.6%). In contrast, only a minor fraction of the patients who had been submitted for PNH testing because of unexplained thrombosis in the absence of cytopenia were positive (3/800; 0.4%).
Conclusion
In summary, our results demonstrate that the current medical indications for PNH screening by FCM are highly efficient, although improved screening algorithms are needed for patients presenting with thrombosis and normal blood cell counts.
Session topic: E-poster
Keyword(s): Diagnosis, Medical patients, Paroxysmal nocturnal hemoglobinuria (PNH)
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