Key Clinical MessagePediatric emergency visits with purpura fulminans should raise the suspicion of hereditary homozygous protein C deficiency even beyond the neonatal age. The absence of this classical finding does not role the diagnosis out as atypical presentation with isolated intraocular bleeding was observed. Premarital counseling should be offered when family history suggests.

No study has been published yet in the Arab world regarding response and outcome of imatinib in patients with chronic myeloid leukemia (CML). This study evaluated a total of 122 patients with CML treated with imatinib between 2001 and 2012. Survival, hematologic, cytogenetic and molecular responses and adverse events were assessed. The 5-year overall survival (OS), event free survival (EFS) and progression-free survival (PFS) rates were: 95.4 ± 2.3%, 81.4 ± 4.6% and 90.8 ± 3.2%, respectively. Significant differences in OS (p = 0.001), EFS (p = 0.001) and PFS (p = 0.001) were noted when patients were stratified by cytogenetic response. Survival by Sokal risk groups was not significant (p = 0.293). Complete hematologic response was achieved in 94 patients (93.1%), cytogenetic response in 84 (83.2%), major molecular response in 62 (61.4%) and complete molecular response in 34 (33.7%). This article presents the first evidence on the effectiveness of imatinib in patients with CML from Saudi Arabia and highlights similarities and differences in response patterns in published studies.

Objectives: To determined the 5-year overall survival (OS) rates for adult patients with acute myeloid leukemia (AML) patients at King Abdulaziz Medical City, Jeddah, Saudi Arabia, based on cytogenetic and molecular abnormalities. Methods: A retrospective cohort study reviewing adult AML patient files between 2011 and 2018. Sixty-three patients were excluded due to pediatric age and secondary AML. The remaining 87 adult patients with de novo AML were enrolled in this study. Results: The most frequent cytogenetic abnormalities were t(15;17) (17.2%), followed by complex cytogenetic (13.8%) and t(8;21) (5.7%). The most frequent molecular abnormalities were promyelocytic leukemia/retinoic acid receptor alpha (PML-RARA) (16.1%) and Nucleophosmin 1 (NPM1) (11.5%). The highest OS rate was associated with t(15;17), PML-RARA, and NPM. However, complex cytogenetic was associated with the lowest OS rate; fms-like tyrosine kinase 3 (FLT3)-internal tandem duplication was independently correlated with low OS rate. Conclusion: The study describes cytogenetic and molecular abnormalities observed in adult AML patients and gives an overview of prognostic factors and determine the OS, with comparable results with recent published data by the WHO.

e18527 Background: Studies have shown superior outcomes of AYA Ph-ALL when treated with pediatric or pediatric-inspired protocol. Since 2003 we used a modified Children’s Oncology Group (COG) protocol to treat AYA Ph-ALL aiming to improve outcomes. Methods: This study involved 67 consecutive patients aged 14-35 years with Ph-ALL diagnosed between October 2003 and March 2016. Patients with precursor B ALL, CNS1/CNS2, and negative day 29 minimal residual disease (MRD-) received single interim maintenance (IM) and delayed intensification (DI) (Arm B). While CNS3 or MRD+ patients received double IM and DI (Arm C). Patients with T cell ALL received double IM with high dose methotrexate (HDMTX) in IM#1, and double DI (Arm C+HDMTX). Dexamethasone was used in all patients but prophylactic cranial irradiation was not utilized. Results: Median age at diagnosis was 17 years with 24 (35.8%) 18 years and older. CR rate, induction death, relapse rate, 4 year event free survival (EFS) and overall survival (OS) were 93.7%, 4.5%, 19.4%, 71.4%±6.0 and 81.8%±5.0; respectively. Of the 57 patients with available MRD, 44 (77.2%) were MRD-. Day 29 MRD status significantly impacted the outcomes (EFS: 78.6%±6.8 vs. 49.2%±15.4; p = 0.007 and OS: 90.5%±4.5 vs. 49.4%±22.0; p = 0.04) for MRD- vs. MRD+ patients; respectively). Outcomes were not different for T (n = 21) vs. B (n = 46) phenotypes (EFS: 69.2%±7.6 vs. 75.6%±9.5; p = 0.57 and OS: 79.8%±6.5 vs. 85.7%±7.6; p = 0.98; respectively). Grade 3 and above toxicities were venous thromboembolism (VTE) in 9 (13.4 %), avascular necrosis (AVN) in 9 (13.4 %) and pancreatitis in 3 (4.5 %) patients. Four patients (6%) died in CR and 2 died with secondary AML. Conclusions: Improved outcomes were observed with OS rate exceeding 90% for MRD negative patients. Therapy was tolerated relatively well, however, AVN and VTE occurred frequently. This suggests that utilizing a modified COG backbone in AYA Ph-ALL patients up to 35 years of age is feasible, however, further modification is warranted to reduce toxicity and improve outcomes in MRD positive patients.

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Inconsistency in prognostic scores occurs where two different risk categories are applied to the same chronic myeloid leukemia (CML) patient. This study evaluated common scoring systems for identifying risk groups based on patients' molecular responses to select the best prognostic score when conflict prognoses are obtained from patient profiles. We analyzed 104 patients diagnosed with CML and treated at King Abdulaziz Medical City, Saudi Arabia, who were monitored for major molecular response (achieving a BCR-ABL1 transcript level equal to or less than 0.1%) by Real-Time Quantitative Polymerase Chain Reaction (RQ-PCR), and their risk profiles were identified using Sokal, Hasford, EUTOS, and ELTS scores based on the patients' clinical and hematological parameters at diagnosis. Our results found that the Hasford score outperformed other scores in identifying risk categories for conflict groups, with an accuracy of 63%.

Whole blood donation has immunomodulatory effects, and most of these have been observed at short intervals following blood donation. This study aimed to investigate the impact of whole blood donation on lymphocyte subsets over a typical inter-donation interval. Healthy male subjects were recruited to study changes in complete blood count (CBC) (n = 42) and lymphocyte subsets (n = 16) before and at four intervals up to 106 days following blood donation. Repeated measures ANOVA were used to compare quantitative variables between different visits. Following blood donation, changes in CBC and erythropoietin were as expected. The neutrophil count increased by 11.3% at 8 days (p < .001). Novel changes were observed in lymphocyte subsets as the CD4/CD8 ratio increased by 9.2% (p < .05) at 8 days and 13.7% (p < .05) at 22 days. CD16-56 cells decreased by 16.2% (p < .05) at 8 days. All the subsets had returned to baseline by 106 days. Regression analysis showed that the changes in CD16-56 cells and CD4/CD8 ratio were not significant (Wilk's lambda = 0.15 and 0.94, respectively) when adjusted for BMI. In conclusion, following whole blood donation, there are transient changes in lymphocyte subsets. The effect of BMI on lymphocyte subsets and the effect of this immunomodulation on the immune response merit further investigation.