Children's Cancer Hospital Egypt Research Articles

Hematopoietic stem cell transplantation from HLA-matched sibling donors in children with acute lymphoblastic leukemia: A report from the Children's Cancer Hospital Egypt.

IntroductionAllogeneic hematopoietic stem cell transplantation (HSCT) is widely used for high-risk acute lymphoblastic leukemia (ALL) patients in their first complete remission (CR1), and for relapsed patients in second complete remission (CR2).Patients and methodsWe retrospectively analyzed data for 67 children with ALL, from a cancer center in a low/middle income country, who had undergone HSCT from human leukocyte antigen (HLA)-matched sibling donors (MSDs) using myeloablative conditioning (MAC) regimens, between 2007 and 2020, describing the survival outcome and relapse probability after achieving CR1 and CR2 and determining outcome differences in relation to indications for HSCT in patients transplanted in CR1. All patients had achieved a negative minimal residual disease prior to transplant (<0.01%).ResultsForty-six patients (68.7%) were in CR1; 25 had adverse cytogenetics, including 18 patients with Philadelphia chromosome-positive ALL (Ph-positive ALL), and 21 had poor induction response. The 5-year overall survival (OS), event-free survival (EFS) and cumulative incidence of relapse (CIR) for the whole cohort were 56.1% (95% CI, 42.8%-69.4%), 49% (95% CI, 35.7%-62.3%) and 33.5% (95% CI, 21.7%-45.8%), respectively with better EFS and CIR for CR1 transplants compared to CR2 transplants (P=0.02 and P=0.03, respectively). Patients with Ph-positive ALL had better 5-year OS, EFS and non-relapse mortality (NRM) compared with other CR1 transplants (P=0.015, P=0.009 and P=0.028, respectively).ConclusionHematopoietic stem cell transplantation from MSD for ALL in CR1 group had superior outcomes compared to CR2 group and was apparently a curable option for Ph-positive ALL without an increased risk of non-relapse mortality. Poorer survival rates and higher relapse probabilities were associated with HSCT conducted to patients who had a poor response to induction therapy or suffered a relapse.

ALL-206 Management and Outcome of Coronavirus Disease 2019 (COVID-19) in Pediatric Cancer Patients: A Single Centre Experience from a Developing Country

Context: Few developing countries have described the impact of coronavirus disease 2019 (COVID-19) on pediatric cancer patients. Most patients had a favorable outcome. The potential benefits of remdesivir (RDV) in pediatric oncology patients require further studies. Objective: Describe the management and outcome of COVID-19 in pediatric oncology patients. Design: Conducted from May to November 2020, this study included pediatric oncology patients with confirmed COVID-19. RDV was the antiviral therapy used. Setting: Pediatric oncology patients were treated at Children's Cancer Hospital Egypt, a specialized cancer center for pediatric oncology. Patients or Other Participants: This prospective study recruited 76 pediatric oncology patients with confirmed COVID-19 infection. Interventions: Allplex2019-nCoV Assay (Seegene, Seoul, South Korea) was used for Multiplex real-time PCR detection of SARS-CoV-2 according to manufacturer instructions. Main Outcome Measures: This study described the clinical course and management of SARS-CoV-2 infections in 76 pediatric oncology patients, detailing disease severity, duration to achieve a negative RT-PCR test, modifications made to protocols, and survival outcomes in patients who had been treated with RDV and those treated without it. Results: The median age of patients was 9 years. Sixty patients were on first-line treatment. Hematological malignancies constituted 86.8% of patients. Severe to critical infections affected 35.4% of patients. The most common symptom was fever (93.4%). Chemotherapy was delayed in 59.2% of patients and doses were modified in 30.2%. The 60-day overall survival (OS) stood at 86.8%, with mortalities occurring only among critical patients. Of 16 acute leukemia patients in the first induction therapy, 13 survived and 10 achieved complete remission. A negative RT-PCR within 2 weeks and improvement of radiological findings were statistically related to disease severity (P=.008 and .002, respectively). Better OS was associated with regression of radiological findings 30 days after infection (P=.002). Forty-five patients received RDV, and 42.1% had severe and critical forms of infection compared to 25.7% in the no-RDV group, yet OS was comparable in both groups. Conclusions: Most pediatric cancer patients with COVID-19 should have good clinical outcomes, except for patients with critical infections. Cancer patients can tolerate chemotherapy, including induction phase, alongside COVID-19 treatment. In severe and critical COVID-19, RDV might have a potential benefit.

Vaginal Tumors in the Pediatric Age Group: The Children's Cancer Hospital Egypt (CCHE)-57357 Experience

Vaginal Tumors in the Pediatric Age Group: The Children's Cancer Hospital Egypt (CCHE)-57357 Experience

RARE-18. Pediatric craniopharyngioma; single center experience in 246 cases with different management modalities

PURPOSE: To report our experience with different craniopharyngioma management strategies done for 246 patients in a single institution during a period of 14 years. METHODS: The medical records of all children with the diagnosis of craniopharyngioma treated at Children's Cancer Hospital Egypt (CCHE-57357) during the period from July 2007 to December 2021 were retrospectively reviewed. RESULTS : our registry included 246 pediatric craniopharyngiomawith median age of 7.4 years old. The main strategies of management after initial surgery were follow up, radiotherapy or administration of intracystic interferone post ommaya insertion. The number of cases in each group were 92, 147,and three respectively. Three patients were not operated upon because of heavily calcified lesions, two of them received radiotherapy while the third was kept under follow up. Overall gross or near total total excision was achieved in 31.2 % , subtotal resection was the case in 42.1% while Ommaya insertion and biopsy was done in 21.1 % of cases. Total number of patients received radiotherapy initially or on progression was 195 patients( 78.9%). The five-year overall survival was 88% (95% CI 82.9 – 93.1) while 5- year progression free survival (PFS) was 49.2% (95% CI 41.2—57.2). The five-year PFS rates for patients in the follow up group versus radiotherapy group were 25.2% and 68.7% respectively (P< 0.0001). Beta catenin was positive in 76.4% of cases that were available for testing (123/161). CONCLUSION: management of craniopharyngioma should be individualized with the main objective is the quality of life. Conservative surgery which entails gross total safe resection whenever possible or lesser extent of resection followed by radiotherapy is the main strategy followed in our institution.

Neuroblastoma-associated Opsoclonous Myoclonous Ataxia Syndrome: Profile and Outcome Report on 15 Egyptian Patients.

Opsoclonous myoclonous ataxia syndrome (OMAS) is a rare primarily immune-mediated disease in children. The current study aim was to find out the patterns and outcome of OMAS associated with neuroblastoma (NBL) among Children's Cancer Hospital-Egypt patients. Data was reviewed for 15 eligible patients enrolled between 2007 and 2016. OMAS treatment included prednisolone and cyclophosphamide with/without intravenous immunoglobulin; NBL treatment was given according to risk-corresponding protocol. Patients' age ranged from 0.75 to 12 years at presentation with male/female: 1.1/1. Concurrent diagnosis of OMAS and NBL occurred in 6 patients (40%). OMAS preceded NBL within 0.25 to 2 years in 33%, while NBL preceded OMAS within 0.5 to 1.5 years in 27%. Full OMAS picture was present in 10/15 patients, while 20% presented with truncal ataxia and myoclonus, 1 with truncal ataxia and opsoclonus, and 1 had opsoclonus and myoclonus. Median time till improvement of manifestations was 5 months. The 5-year OMAS progression-free survival was 33.3%, where 10 patients needed second-line therapy due to relapse/progression of OMAS. The median time to progression was 28 months measured from OMAS diagnosis. All patients remained alive with NBL 5-year overall survival of 100% and event-free survival of 85.7% for. However, 73% of the patients showed late sequelae ranging from ocular to cognitive, behavioral and motor disorders; rarely seizures and hemolytic anemia.

Prognostic implication of serum Alpha-fetoprotein response to neoadjuvant chemotherapy in Hepatoblastoma patients

Objective: This retrospective study aims to identify the early changes in serum alpha-fetoprotein levels (AFP) and their correlation with the survival outcome of hepatoblastoma patients. Materials and Methods: A total of 68 patients presented to the children's cancer hospital Egypt and the national cancer institute from January 2013 till June 2016 were included in this study. Results: AFP level was measured post-cycle 2 in 60 patients; 44 (73.3%) patients showed a decline in AFP level by &gt;1 log reduction. The 3-year EFS was 75.6% for patients with &gt;1 log reduction in AFP level, compared with 36.5% for those with &lt;1 log reduction (p=0.010). The 3-year OS’ for patients with &gt;1 and &lt;1 log reduction in AFP level were 80.4% and 39.4%, respectively (p=0.005). On multivariate analysis; Patients with AFP log reduction&lt; 1 had worse OS/EFS with hazards ratio (HR): 3.9 and 95% confidence interval (CI):1.4-11.2, p value=0.011 and HR: 3.2 and 95% CI: 1.3-8.9, p value=0.013 respectively. Conclusion: The ease of AFP determination makes it a valuable tool that could be routinely used to evaluate the therapeutic efficacy and predict the survival outcome.

Association of Day +100 Platelet Count and Survival Post Allogenic Hematopoietic Stem Cell Transplantation

Introduction :Recovery of platelet count by day 100 post allogeneic HSCT is reported to be a predictor of overall survival. Delayed platelet recovery could impact outcome due to its association with incipient disease relapse, graft failure, infection or other causes of treatment related mortality (TRM).We investigated the correlation between day +100 platelet count and overall survival post allogeneic HSCT in pediatric cancer patients.Methods:This was a retrospective study of consecutive patients who underwent allogeneic HSCT at the Children Cancer Hospital Egypt (CCHE 57357) between 2009 and 2015 with a minimum follow up duration of one year post transplant. All eligible patients received myeloablative conditioning. All patients had matched related donors, except one with an unrelated cord blood donor. Patients who survived without relapse until day 100 post HSCT were divided into two groups: early platelet recovery (EPR) (platelet count >100x109/L at day +100 post transplant) and delayed platelet recovery (DPR) (platelet count ≤ 100x109/L at day +100 post transplant).Results :During the study period, 155 patients were transplanted for hematological malignancies: AML (58), ALL (38), mixed phenotype leukemia (5), CML (33), JMML (14), MDS (5), NHL (2). The median age at transplant was 10 years. Stem cell source was bone marrow in 92 patients (59.4%), peripheral blood stem cells in 58 patients (37.4%) and cord blood in 5 patients (3.2%) with median CD34+ stem cell dose 5.6 x 106/kg.At day+100, 40 patients (26%) had DPR, 113 patients (74%) had EPR (two patients were excluded: one died before day+100 and one was lost to follow-up). With a median follow-up of 41 months (Range 1 - 93 months), 43 patients (27.7%) died, 36 due to relapse, 6 from TRM and one unknown. The 3 year disease free survival (DFS) and overall survival (OS) was 68±7.84 % and 71.9±7.84 % respectively. For EPR patients, 25 (22%) died compared to 17 (42.5%) DPR patients. The 3 year overall survival was 77.9% and 57.1% for the EPR and DPR respectively (p 0.006). The 3 year DFS of the EPR and DPR groups were 73.2±9% and 54.8±16.3% respectively (p 0.02). Incidence of disease relapse for EPR and DPR patients was 22.6 %(95%CI 15.1-31%) and 39.5(95% CI 23.7 - 54.8%) respectively (p 0.04). Multivariate analysis for survival identified delayed platelet recovery as a predictor of decreased survival (p 0.002) (HR 3.23 95% CI 1.56-6.66). Chronic GVHD, was also a significant predictor of decreased survival (p 0.044) (HR 2.54 95% CI 1.03-6.31).Conclusion:Platelet count at Day 100 is simple to obtain, inexpensive and routinely collected as part of clinical care. Platelet reconstitution is a complicated process and abnormal platelet counts can be due to many different reasons. Our results suggest that patients with a robust platelet count at Day 100 can be considered in a good risk category. However, patients failing to achieve a normal platelet count have inferior long term OS and DFS. Thus thrombocytopenia, regardless of etiology, can be used to identify a vulnerable group of patients who require close observation and may be considered for further diagnostic evaluation. DisclosuresNo relevant conflicts of interest to declare.

Clinical Significance of MicroRNA-29a and MicroRNA-100 Gene Expression in Pediatric Acute Myeloid Leukemia.

The aim of this study was to evaluate the diagnostic and prognostic performance of miRNA-29a and miRNA-100 in pediatric acute myeloid leukemia (AML). In all, 73 children with diagnosed pediatric AML (based on standard morphologic, cytochemical, cytogenetic, immunologic, and molecular workup, and the French-American British classification) admitted to Children's Cancer Hospital Egypt (CCHE-57357), and 9 healthy age-matched and sex-matched controls were recruited for a case-control study. Gene expression levels of miRNA-29a and miRNA-100 were assessed using real-time quantitative RT-PCR. When diagnosed, patients had a significantly higher expression of miRNA-100 as against controls (median [range]: 12.99 [0.92-851.38] vs. 0.26 [0.03-2.67], P<0.001), with a significantly lower expression of miRNA-29a (2.08 [0.02-19.72] vs. 24.95 [15.48-42.54], P<0.001). Likewise, high-risk patients according to cytogenetic stratification had significantly higher miRNA-100 expression and lower miRNA-29a expression. Both miRNA-100 and miRNA-29a performed well as diagnostic markers of pediatric AML with an area under the curve of 0.977 (95% confidence interval [95% CI: 0.943-1.0]) and 0.994 (0.982-1.0) for miRNA-100 and miRNA-29a, respectively. Both miRNA-29a (odds ratio [95% CI]: 0.160 [0.054-0.474], P=0.001) and miRNA-100 (odds ratio [95% CI]: 1.997 [1.994-2.001], P=0.047) were identified as significant predictors of treatment response. The miRNA-29a and miRNA-100 expression may serve as diagnostic and prognostic markers in pediatric AML.

Cardiovascular and Thyroid Late Effects in Pediatric Patients with Hodgkin Lymphoma Treated with Abvd Protocol

Background: Long-term Hodgkin Lymphoma (HL) survivors are at risk of developing a range of therapy-related complications that may present 10 -15 years after treatment. The goal of this study is to estimate the frequency of cardiovascular diseases and thyroid abnormalities in HL survivors and to determine the contributing risk factors. Methods: We performed a cross-sectional study on 208 HL survivors who were treated at the National Cancer Institute, Cairo University, or at the Children Cancer Hospital Egypt with ABVD chemotherapy ± radiotherapy with a minimum of five years follow up. Findings: The cumulative incidence of cardiac toxicity (CT) at five and nine years were 18·7% ± SD 2·7 and 43·3% ± SD 4·4, respectively. Preexisting cardiac abnormalities, cumulative anthracycline dose, and end of treatment cardiac status are strong predictors for late cardiotoxicity. Hypertension was observed in about 31% of HL survivors. Young age and obesity at the time of treatment are important factors for hypertension. Thyroid abnormalities developed with a 5-year cumulative incidence of 2%± SD 1 while at 9-years the cumulative incidence was 27·9%± SD 4·5. Thyroid dysfunction was observed in 21·2% and thyroid tumors in 1·6%. Subclinical hypothyroidism was the most common thyroid abnormality after HL treatment. Neck radiation therapy and the volume of thyroid gland exposed to radiation are important risk factors. Interpretation: Cardiotoxicity, hypertension, and thyroid dysfunction are frequent late effects following the ABVD regimen especially if combined with radiation therapy. Funding: None to declare. Declaration of Interest: The authors declare that there is no conflict of interest. Ethical Approval: The study was approved by the institutional review board of the NCI-Cairo University and the scientific medical advisory committee of CCHE.

Benefits of endoscope-assisted microsurgery in the management of pediatric brain tumors.

Microsurgical and endoscopic techniques are vastly utilized in brain tumor surgery. Combining both techniques in the same procedure has different forms and applications. The aim of this work was to discuss the usefulness and describe the technical benefits of endoscope-assisted microsurgery (EAMS) in treating pediatric brain tumors in various anatomical locations. The medical records of 106 children who had undergone EAMS for brain tumors at Children's Cancer Hospital Egypt (CCHE-57357) between January 2009 and January 2017 were reviewed. The patients' ages ranged from 1 to 16 years (mean age 7.5 years). Technical variations, difficulties, complications, strategies, and extent of resection were addressed according to anatomical location. In general, EAMS enabled closer inspection of tumor extension and surrounding vital structures, especially in the hidden corners not appreciable by the microscope alone, such as tumors in the internal auditory canal and cerebellopontine angle contents in 14 cases, all of which were totally excised, and the undersurface of the optic apparatus in 65 craniopharyngiomas. Total excision was achievable in 51 of the 65 craniopharyngiomas; residual tumor was intentionally left behind under endoscopic guidance in the remaining 14 patients to ensure better hypothalamic function. Vision improved in 15 of 16 patients who initially presented with visual defects. Only 4 patients had new-onset postoperative endocrinopathies. For intraventricular tumors, EAMS allowed earlier recognition of tumor pedicle and, hence, earlier control of the blood supply of the tumor and safer total excision of 12 lateral ventricle, 6 pineal and third ventricle, and 9 fourth ventricle tumors. The tandem use of the endoscope and microscope enabled safer tumor dissections that were performed with more confidence in situations in which pure microscopic excision was either not achievable or less safe. Technical strategies, pitfalls, difficulties, and precautions were categorized and described per tumor location. EAMS of pediatric brain tumors is a promising, user-friendly tool that complements microsurgery in the management of these complex lesions. The benefits of 2D endoscopy are added to the benefits of stereoscopic perception. EAMS is especially helpful during the removal of different complex pediatric brain tumors. Simultaneous or tandem endoscopic and microscopic approaches may have the potential for better functional outcomes through better visualization and preservation of vital structures in corners that are hidden from the microscope.

Patterns, risk factors and outcome predictors of posterior reversible encephalopathy syndrome in pediatric cancer patients

The purpose of this study was to assess the clinical and radiological patterns and outcome predictors of posterior reversible encephalopathy syndrome (PRES) in pediatric cancer patients. A retrospective study included patients who developed PRES during their treatment at the Children's Cancer Hospital Egypt. A total of 50 patients developed PRES. Leukemia and lymphoma were the commonest diagnoses (64%). Regarding the MRI findings, occipital affection was the most common (92%), followed by frontal and temporal lobes involvement in 32% and 22% respectively and advanced PRES was described in 8 patients. Of the whole patients, 80% had complete clinical resolution and 60% showed complete radiological resolution at 2 weeks’ evaluation and 2 patients died out of PRES. Unfavorable outcome was associated with those who had motor dysfunction, status epilepticus at presentation, frontal lobe and thalamic affection and atypical PRES. PRES might present in atypical sites with poor outcome including death.

Temporal trends in childhood cancer survival in Egypt, 2007 to 2017: A large retrospective study of 14 808 children with cancer from the Children's Cancer Hospital Egypt.

Childhood cancer is a priority in Egypt due to large numbers of children with cancer, suboptimal care and insufficient resources. It is difficult to evaluate progress in survival because of paucity of data in National Cancer Registry. In this study, we studied survival rates and trends in survival of the largest available cohort of children with cancer (n = 15 779, aged 0-18 years) from Egypt between 2007 and 2017, treated at Children's Cancer Hospital Egypt-(CCHE), representing 40% to 50% of all childhood cancers across Egypt. We estimated 5-year overall survival (OS) for 14 808 eligible patients using Kaplan-Meier method, and determined survival trends using Cox regression by single year of diagnosis and by diagnosis periods. We compared age-standardized rates to international benchmarks in England and the United States, identified cancers with inferior survival and provided recommendations for improvement. Five-year OS was 72.1% (95% CI 71.3-72.9) for all cancers combined, and survival trends increased significantly by single year of diagnosis (P < .001) and by calendar periods from 69.6% to 74.2% (P < .0001) between 2007-2012 and 2013-2017. Survival trends improved significantly for leukemias, lymphomas, CNS tumors, neuroblastoma, hepatoblastoma and Ewing Sarcoma. Survival was significantly lower by 9% and 11.2% (P < .001) than England and the United States, respectively. Significantly inferior survival was observed for the majority of cancers. Although survival trends are improving for childhood cancers in Egypt/CCHE, survival is still inferior in high-income countries. We provide evidence-based recommendations to improve survival in Egypt by reflecting on current obstacles in care, with further implications on practice and policy.

Management of pediatric craniopharyngioma: 10-year experience from high-flow center.

To report our experience and management strategies during 10years for 137 childhood craniopharyngiomas treated at a single institution. Medical records of children with craniopharyngioma treated at Children's Cancer Hospital Egypt (CCHE-57357) from July 2007 to December 2017 were retrospectively reviewed. Beta-catenin as an immunohistochemical marker was assessed also in available specimens. Our registry included 137 patients. Headache (n = 122), visual failure (n = 118), and hypothyroidism(n = 78) were the most common findings on presentation. Three management protocols were identified; 65 patients were primarily followed up after surgery, 71 patients had radiotherapy after surgery, and one patient underwent surgery for Ommaya insertion with intracystic interferon injection. Overall, gross total resection/near total resection was achieved in 48 cases (35.04%), subtotal resection was achieved in 58 patients (42.33%), 29 (21.16%) had biopsy and Ommaya reservoir, and two patients with calcified lesions had no operations. Fifty-four patients showed recurrence/progression of their lesions. Allover, 5-year progression-free survival (PFS) was 52.3%, while it was 34.49% and 72.25% for the follow-up group and the radiotherapy group, respectively. Beta-catenin mutations were positive in 61/95 patients; 5-year PFS for beta-catenin negative and positive cases was 65.5% and 39.4% respectively (p = 0.087). Mortality was reported in eight patients. Intraoperative endoscopy-assisted assessment was the cornerstone of tailored decision-making. The concepts of conservative surgery and multimodal management should be applied to reach the perfect balance between the quality of life and the best tumor control rates. Beta-catenin mutations more than 5% are associated with statistically trending aggressive clinical behavior. The CCHE-57357 algorithm of individualized management protocol was presented.

Can FDG-PET replace biopsy for the evaluation of residual tumor in pediatric mature B-cell non-Hodgkin lymphoma?

The aim of our study is to evaluate the role of 18 F-labeled fluorodeoxy glucose positron emission tomography (18 FDG-PET) scan for the detection of viable residual mass in pediatric mature B-cell non-Hodgkin lymphoma (NHL). This study also aims to detect the negative predictive value, positive predictive value (PPV), sensitivity, and specificity of 18 FDG-PET. A retrospective, cross-sectional nonrandomized study was carried out. We included all patients with newly diagnosed mature B-cell NHL treated at the Children Cancer Hospital Egypt during the period between July 2007 and the end of May 2018. Patients were included in the study if they (a) had a residual tumor mass, (b) underwent an 18 FDG-PET scan, and (c) had a pathologic documentation of this residual tumor. Patients were followed up till June 2019. Thirty-six patients were included, for whom 39 biopsies were performed. Mean age was 7.7 years. Median follow-up period was 52.8, range 6.1 to 117 months.18 FDG-PET scan was positive (Deauville score 3, 4, or 5) in 24 of 39 patients (61.5%), while it was negative (Deauville score 1 or 2) in 15 patients (38.5%). Positive 18 FDG-PET scan and biopsy were performed in 15 of 39 samples (38.4%; true positive, TP), while they were both negative in 13 samples (33.3%; true negative). Nine patients (23%) had positive scan and a negative biopsy (false positive), while 2 patients had negative uptake and a positive biopsy (false negative, FN)). Sensitivity of the 18 FDG-PET scan was 88.2% and specificity was 59.1%. PPV was 62.5% and NPPV was 86.6%. Changing therapy on the basis of a positive finding alone at the time of evaluation is not recommended. FN results exist, so biopsy confirmation is required to avoid the missing refractory disease. If negative, 18 FDG- PET can replace a biopsy if the latter is inaccessible or carries an unnecessary risk.

Assessment of Healthcare Professionals’ Knowledge, Attitudes, and Perceived Challenges of Clinical Pharmacogenetic Testing in Egypt

Aim: We evaluated healthcare practitioners'perspectives regarding clinical pharmacogenetics in Cairo, Egypt. Materials & methods: We administered a paper-based survey to pharmacists and physicians practicing at Children's Cancer Hospital Egypt. The survey assessed practitioners' knowledge, attitudes, and perspectives about pharmacogenetic testing. Results: The study included 184 respondents (67.9% pharmacists; 32.1% physicians. Overall, the pharmacogenetic knowledge was low (mean=41.7%) but attitudes toward pharmacogenetic testing and its potential clinical application were generally positive. Pharmacists responded more favorably than physicians to statements attributing the responsibility of applying pharmacogenetics in the clinical setting to their profession. However, several challenges were identified; the most common being: lack of pharmacogenetic knowledge and skill, lack of pharmacogenetic testing devices, and limited funding. Conclusion: Future efforts to promote pharmacogenetic implementation should focus on foundational education, practical training, and exploration of potential funding sources.

Outcome of Children Treated for Infantile Hepatic Hemangioendothelioma.

Infantile hepatic hemangioendothelioma (IHHE) is the most common hepatic vascular tumor in children. We report on the treatment outcome of our large single-center experience of patients with IHHE over a 9-year period. A retrospective analysis of all IHHE patients treated at the Children Cancer Hospital Egypt from April 2008 through April 2017. In total, 28 patients (18 females, 10 males) were diagnosed with IHHE with a median age at diagnosis of 3 months. The lesions were multifocal (n=12), focal (n=10), and diffuse (n=6). Six (21.4%) patients initially had low T3 and T4. Eleven patients did not receive any treatment, whereas 1 patient underwent resectional surgery. Sixteen patients received drug treatment, 9 of whom responded well to first-line propranolol/prednisolone, whereas 7 patients needed salvage treatment. Twenty-five patients are alive, whereas 3 patients have died. Overall, patients with IHHE do well, a significant percentage of whom do not require drug therapy, particularly for those with small focal lesions. In patients with multifocal/diffuse disease, there is a high incidence of low T3 and T4 and while some of these patients did well without additional therapy, those with rapidly progressive lesions during treatment may do poorly.

Outcomes of allogenic hematopoietic cell transplantation for childhood chronic myeloid leukemia: Single‐center experience

Despite the apparent efficacy and favorable toxicity profile of TKIs, allogeneic SCT remains the only curative treatment for CML especially in younger patients, but TRM should be considered. We evaluated the clinical outcomes of pediatric CML patients who had SCT in our center. This retrospective study included children with CML, who received an allogeneic SCT at Children Cancer Hospital Egypt, 57357, from 2007 to 2017. All patients received myeloablative conditioning chemotherapy containing busulfan/cyclophosphamide followed by stem cell infusion from MRD. From 121 patients diagnosed with CML, 43 had available MRD and subjected to HSCT while 78 patients continued TKI therapy. The median time to transplant from diagnosis was 13months. At initial diagnosis, there were 39 patients in CP and 4 had blastic crises. Bone marrow harvest was the stem cell source in 32 patients, while 11 cases received mobilized peripheral blood stem cells with average stem cell dose of 4.45×106 /kg. The probabilities of overall survival and event-free survival at 5years were 97.4% and 79.8%, respectively. TRM at 100days and TRM at 1-year post-transplant were 0%. The incidence of chronic GVHD was significantly higher in peripheral blood than bone marrow stem cell source (P=.004). Considering the excellent survival rates and very low TRM, HSCT is still a valid option for pediatric patients with newly diagnosed CML with best using marrow stem cell source to avoid a significant risk of cGVHD and its related complications.

Clinical features and outcome of hepatosplenic fungal infections in children with haematological malignancies

Hepatosplenic fungal infection (HSFI) is a severe invasive fungal infection observed during neutrophil recovery in patients with acute leukaemia treated with intensive chemotherapy. Retrospective analysis including all paediatric haematological malignancies patients with HSC treated in Children Cancer Hospital Egypt (2013-2018). Twenty-five patients with acute leukaemia developed HSFI (19 patients diagnosed as hepatosplenic candidiasis). Most of the cases (92%) occurred during the induction phase. Organs affected were as follows: liver in 18 patients, renal in 13 patients, spleen in 12 patients, skin in four patients and retina in one patient. Five (20%) patients had proven HSC, 14 (56%) probable and six (24%) possible HSFI. Ten patients had a PET-CT for response assessment. Candida tropicalis was the most common isolated spp. from blood/tissue culture. Six (24%) patients developed HSFI on top of antifungal prophylaxis. Steroids were given in 12 (52%) patients with HSFI as immune reconstitution syndrome (IRS). Caspofungin was the first line of treatment in 14 (56%) patients, liposomal amphotericin B in six (24%) patients and azoles in five (20%) patients. HSFI was associated with delayed of intensification phase of chemotherapy (median 42days). The success rate was reported in 24 patients with complete response (68%) and partial response in (28%) patients, while failure (death) seen in 1(4%) patient. HSC is still a major challenge in paediatric leukaemias patients with impact on treatment delay and survival outcome. PET scan, non-culture diagnostics and steroid role evidence in IRS are growing. Antifungal stewardship for screening, early detection for high-risk patients and better response assessment is challenging.

Pretreatment Risk Factors Is a Strong Predictor of Outcome in Pediatric Hodgkin Lymphoma

Background: During the last decades, the prognosis of Hodgkin lymphoma (HL) has been improved significantly with the introduction of effective chemotherapy and the implementation of risk-adapted treatment approaches. Identification of reliable risk factors is crucial to guide treatment throughout the disease. Objectives: We aimed to identify independent predictors of event-free survival (EFS) in pediatric/ adolescent HL using clinical data known at diagnosis and treated with the ABVD regimen at our hospital. Design/Method: This is a retrospective study done at Children's cancer hospital Egypt from July 2007 to December 2017. ALL Patients with Classical HL were enrolled in the analysis. The chemotherapy backbone was ABVD +/- RTH. Data analysis included all pretreatment Demographics, clinicopathological characteristics, and Laboratory findings. PET scan was performed at baseline and after two cycles of chemotherapy. Treatment was not changed according to the results of the interim scan. Results: A total of 737 patients were eligible for analysis.The Five-years OS 94.9% and the EFS 87.8% in these patients. Pretreatment Significant univariate predictive factors for the EFS were: Age&lt;9.5 (HR 0.421; p = 0.002),, ESR (HR 0.421; p = 0.002), Hemoglobin &lt;10.5 (HR 1.373; p = 0.014), TLC &gt;11,000 (HR 0.368; p = 0.000), Stage IV( p = 0.000) and treatment groups (HR 0.180; p = 0.000). Although the pathological subtypes has a diverse EFS;NS: 86.6% MS: 92.9% Lymphocyte rich: 87.5% and Lymphocyte depleted: 41.7%, Histology has shown to be a powerful independent predictor of outcome ( p = 0.000).In the multiple regression model, NS Histology and stage remained strong predictive factors with a p-value of 0.011and 0.026 respectively, In addition to the significance of the Albumin level (HR 0.047, p= 0.011). The interim PET, as well as RTH, were highly significant on both univariate (p= 0.000 and 0.0002 respectively) as well as multivariate analysis (p= 0.001and 0.000 respectively). Conclusion: Age; stage IV disease; white blood cell count, and hemoglobin level are independent predictors of survival that can significantly impact survival. Optimizing initial therapy may improve overall EFS for these patients. Although CHL pathology is a significant independent predictor of EFS, its subtypes have different EFS and OS, which should be translated into changes in the treatment approach. Disclosures No relevant conflicts of interest to declare.

2689. Stenotrphomonas maltophilia, The Hidden Threat Among Pediatric Cancer Patients

BackgroundStenotrophomonas maltophilia is an emerging nosocomial pathogen in immunocompromised patients. Although S. maltophilia exhibits limited pathogenicity in immunocompetent hosts, it has been shown to cause fatal infections in patients with malignancies. The objective of this study to analyze the clinical characteristics, susceptibility pattern, and treatment outcome of S. maltophilia among pediatric cancer patients.MethodsRetrospective analysis including all pediatric cancer patients treated at children cancer hospital Egypt (CCHE) with S. maltophilia bloodstream infection from June 2013 till June 2018.Results281 isolates among 135 pediatric cancer patients. Most are hematological malignancies 67(50%), solid tumors 55 (40%) and post-transplant 13(10%). Most common hematological malignancies were acute lymphoblastic leukemia 34 patients (25%) while brain tumor was the most common solid tumors 20 patients (15%). The spectrum of infections includes bacteremia in 61 patients (45%) catheter-related in 34 (25%), pneumonia in 22 (16%), skin and soft-tissue infection in 11(8%) meningitis in 5 (3%) and disseminated infections with multiorgan involvement in 4(3%) patients. 46 patients (34%) was admitted in intensive care unit (ICU), 67 inpatient (50%), 11 (8%) stem cell transplant unit and 11 patient (8%) from emergency and outpatient department. The isolates revealed 80% susceptibility to Trimethoprim-Sulfamethoxazole (TMP-SMX), 77% to ciprofloxacin, 50% to cefepime and ceftazidime, 63% to amikacin, 48% to piperacillin–tazobactam, 93% to colistin, 97% to tigecycline. Day 30 mortality (Crude mortality rate) 33 patients (25%) while S. maltophilia attributable mortality (died within 7 days of culture isolation) was 17 patients (13%). Patients with pneumonia, (TMP-SMX) resistance and ICU admission were associated with a significant risk of mortality.ConclusionStenotrphomonas bloodstream is a serious pathogen and hidden threat among pediatric cancer patients associated with high mortality rate.Disclosures All authors: No reported disclosures.