Predicting response to porcine antilymphocyte globulin plus cyclosporine A in children with acquired severe aplastic anemia

The predictive value of pre-treatment paroxysmal nocturnal hemoglobinuria clone on response to immunosuppressive therapy in patients with aplastic anemia: a meta-analysis

“…After carefully reading the full texts, 26 articles were excluded for insufficient data (n = 22), low sample size (n = 1), and overlapped populations (n = 3). Finally, 12 studies [8,10,12,[14][15][16][20][21][22][23][24][25] were included in this meta-analysis.…”

“…Of the 12 eligible studies, 2 studies were from North America [8,22], 3 from Europe [10,15,23], and 7 from Asia [12,14,16,20,21,24,25]. Five studies [12,16,22,23,25] included pediatric patients only, others included pediatric and adult patients [8,[13][14][15]20,21,23,24]. All the patients in the 12 studies were treated by IST, but therapy information was not available in 2 studies [15,25].…”

“…The criteria of response were also inconsistent. In 10 studies [10,12,[14][15][16]20,[22][23][24][25] the response was defined as complete response (defined as hemoglobin level within normal limits for age, platelet count >100 × 10 9 /L, and absolute neutrophil count >1.5 × 10 9 /L) plus partial response (defined as transfusion independence, absolute neutrophil count >0.5 × 10 9 /L and absolute reticulocyte count >30 × 10 9 /L). In the study conducted by Scheinberg et al [8], the response was defined as no longer meeting criteria for SAA.…”

“…Five studies [16,21,22,24,25] reported the effect of the pre-treatment PNH clone on the overall response to IST in AA. As moderate heterogeneity among the five included studies (I 2 = 61.3%, p = 0.035) was detected, a random-effects model was applied to evaluate the pooled OR.…”

“…With the introduction of flow cytometry (FCM), the pre-treatment PNH clone can be detected in a fraction of AA [10][11][12], though almost asymptomatic. However, its predictive value on the response to IST still remains debatable, and serial studies reported favorable [10][11][12][13][14], neutral [8] and even negative [15,16] results. To further elaborate this issue, we conducted this meta-analysis.…”