A 10-year-old male with chest wall Desmoid tumor - a rare tumor with unusual presentation.

Desmoid fibromatosis (DF) is a connective tissue tumor that grows aggressively in musculoaponeurotic tissues. It has an annual incidence rate of 2-4/million and is commonly seen in individuals aged 15 to 60 years, with female predominance. While it can occur in any body part, it is commonly found in the extremities, abdominal wall, and abdominal mesentery. But it rarely develops in the chest wall. The cause of the tumor is unknown. However, trauma to the tumor site has been identified in 25% of known cases. The primary treatment is surgical resection. Local recurrence after surgical excision is common. Our study highlights the case of a 12-year-old male patient diagnosed with a desmoid tumor on the chest wall 2 years ago, at the age of 10 years, after mild trauma. The tumor was successfully managed with surgery without radiotherapy treatment and no recurrence was observed in the last 2 years.

Unusual initial presentation of childhood acute lymphoblastic leukemia as massive ascites and pleural effusion in post-COVID-19 setting: a case report.

Acute lymphoblastic leukemia (ALL) in children typically presents with nonspecific manifestations such as fever, fatigue, lethargy, joint and bone pain, and bleeding diathesis. Ascites and pleural effusion as an initial presentation of ALL, although described, is exceedingly rare. However, this unusual initial presentation becomes much rarer in the post-coronavirus disease 2019 (COVID-19) setting. Herein, we aim to highlight such a rare initial presentation of childhood ALL that warrants clinical attention. Case Presentation: Two months following a COVID-19 infection, a 3-year-old male patient presented to the hospital with severe abdominal distention associated with occasional dyspnea. Physical assessment revealed a critically ill and pale patient with a distended abdomen and decreased air entry on the right side of the chest. Laboratory testing showed pancytopenia. Imaging studies confirmed the presence of massive ascites and pleural effusion. Bone marrow aspiration revealed CD10-positive pre-B-cell ALL. The patient was treated with chemotherapy and achieved complete remission. Conclusion: Rare manifestations of relatively common diseases create a barrier to prompt and effective detection and medical intervention. Although ascites and pleural effusion are rare conditions in ALL children patients, the occurrence of these pathologies in this particular patient, especially following COVID-19 infection, is an exceedingly rare event.

[STEM CELL TRANSPLANTATIONS FOR PATIENTS WITH FANCONI ANEMIA: AN ISRAELI TERTIARY CENTER EXPERIENCE].

INTRODUCTION: Fanconi anemia (FA) is a rare genetic syndrome characterized by increased chromosomal breakage, congenital anomalies, bone marrow failure and an increased tendency to develop malignancies. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment for bone marrow failure and the hematologic malignancies these patients develop. Given the sensitivity of FA patients to chemotherapy and radiation, as to the clinical symptoms of graft versus host disease (GvHD), HSCT in these patients is challenging. Since the mid-nineties, HSCT for FA patients is performed in our center by using the fludarabine based reduced-intensity protocol. AIMS: To summarize the results of HSCT for patients with FA using a fludarabine based reduced-intensity conditioning regimen at the Hadassah Medical Center. METHODS: This retrospective research is based on the collection and analysis of clinical and laboratory data from the medical records of patients. RESULTS: Since June 1996 up till February 2020, 39 patients with FA underwent 43 HSCTs with a fludarabine based protocol at the Hadassah Medical Center. Four patients required a second transplant due to primary engraftment failure. Nine patients (23%) suffered from acute GvHD, four of them severe. Eight patients (20%) developed chronic GvHD, two with an extensive and debilitating disease. Thirty-three (85%) of the patients survived and six died, five shortly after the transplant, and one twenty years later from malignancy. CONCLUSIONS: Our results show high survival rates with low rates of engraftment failure and reasonable rates of GvHD. DISCUSSION: As of today, there is an effective and safe treatment for patients with FA who require HSCT by using a fludarabine-based reduced-intensity conditioning regimen, with high survival rates and few complications.

Two decades of stem cell transplantation in patients with Fanconi anemia: Analysis of factors affecting transplant outcomes.

Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only curative treatment for the hematological complications of patients with Fanconi anemia (FA). Over the last two decades, HSCT outcomes have improved dramatically following the development of regimens tailored for FA patients. In this study, we analyzed genetic, clinical, and transplant data of 41 patients with FA who underwent HSCT at Hadassah Medical Center between November 1996 and September 2020. Overall survival (OS) was 82.9% with a median follow-up time of 2.11-years (95% CI, .48-16.56). Thirteen patients (31.7%) developed acute graft-versus-host disease (GVHD), three of them with grades 3-4. Nine patients developed chronic GVHD, five had extensive disease. Twelve patients (29.3%) developed stable mixed-chimerism with complete resolution of bone marrow failure (BMF); none of them had acute nor chronic GVHD. Significantly higher GVHD rates were observed in transplants from peripheral blood stem cell grafts as compared to other stem cell sources (p = .002 for acute and p = .004 for chronic GVHD). Outcome parameters were comparable between HSCT from matched-sibling (n = 20) to other donors (n = 21), including survival rates (p = .1), time to engraftment (p = .69 and p = .14 for neutrophil and platelet engraftment time, respectively), chimerism status (p = .36 and p = .83 for full-donor and mixed chimerism, respectively), and GVHD prevalence (p = 1). Our results demonstrate the vast improvements in HSCT outcomes of patients with FA, narrowing the gap between matched-sibling versus alternative donor transplantations. Our data identifies factors that may significantly affect transplant outcomes such as graft source and chimerism status.

Implementation of a pediatric early warning score tool in a pediatric oncology Ward in Palestine.

BACKGROUND: Pediatric Early Warning Scores (PEWS) are nurse-administered clinical assessment tools utilizing vital signs and patient signs and symptoms to screen for patients at risk for clinical deterioration.1-3 When utilizing a PEWS system, which consists of an escalation algorithm to alert physicians of high risk patients requiring a bedside evaluation and assessment, studies have demonstrated that PEWS systems can decrease pediatric intensive care (PICU) utilization, in-hospital cardiac arrests, and overall decreased mortality in high income settings. Yet, many hospital based settings in low and lower middle income countries (LMIC) lack systems in place for early identification of patients at risk for clinical deterioration. METHODS: A contextually adapted 16-h pediatric resuscitation program included training of a PEWS tool followed by implementation and integration of a PEWS system in a pediatric hematology/oncology ward in Beit Jala, Palestine. Four PDSA cycles were implemented post-implementation to improve uptake and scoring of PEWS which included PEWS tool integration into an existing electronic medical record (EMR), escalation algorithm and job aid implementation, data audits and ward feedback. RESULTS: Frequency of complete PEWS vital sign documentation reached a mean of 89.9%. The frequency and accuracy of PEWS scores steadily increased during the post-implementation period, consistently above 89% in both categories starting from data audit four and continuing thereafter. Accuracy of PEWS scoring was unable to be assessed during week 1 and 2 of data audits due to challenges with PEWS integration into the existing EMR (PDSA cycle 1) which were resolved by the 3rd week of data auditing (PDSA cycle 2). CONCLUSIONS: Implementation of a PEWS scoring tool in an LMIC pediatric oncology inpatient unit is feasible and can improve frequency of vital sign collection and generate accurate PEWS scores. CONTRIBUTION TO THE LITERATURE: This study demonstrates how to effectively implement a PEWS scoring tool into an LMIC clinical setting. This study demonstrates how to utilize a robust feedback mechanism to ensure a quality program uptake. This study demonstrates an effective international partnership model that other institutions may utilize for implementation science.

Impact of the coronavirus disease 2019 (COVID-19) pandemic on pediatric oncology care in the Middle East, North Africa, and West Asia region: A report from the Pediatric Oncology East and Mediterranean (POEM) group.

BACKGROUND: Childhood cancer is a highly curable disease when timely diagnosis and appropriate therapy are provided. A negative impact of the coronavirus disease 2019 (COVID-19) pandemic on access to care for children with cancer is likely but has not been evaluated. METHODS: A 34-item survey focusing on barriers to pediatric oncology management during the COVID-19 pandemic was distributed to heads of pediatric oncology units within the Pediatric Oncology East and Mediterranean (POEM) collaborative group, from the Middle East, North Africa, and West Asia. Responses were collected on April 11 through 22, 2020. Corresponding rates of proven COVID-19 cases and deaths were retrieved from the World Health Organization database. RESULTS: In total, 34 centers from 19 countries participated. Almost all centers applied guidelines to optimize resource utilization and safety, including delaying off-treatment visits, rotating and reducing staff, and implementing social distancing, hand hygiene measures, and personal protective equipment use. Essential treatments, including chemotherapy, surgery, and radiation therapy, were delayed in 29% to 44% of centers, and 24% of centers restricted acceptance of new patients. Clinical care delivery was reported as negatively affected in 28% of centers. Greater than 70% of centers reported shortages in blood products, and 47% to 62% reported interruptions in surgery and radiation as well as medication shortages. However, bed availability was affected in <30% of centers, reflecting the low rates of COVID-19 hospitalizations in the corresponding countries at the time of the survey. CONCLUSIONS: Mechanisms to approach childhood cancer treatment delivery during crises need to be re-evaluated, because treatment interruptions and delays are expected to affect patient outcomes in this otherwise largely curable disease.

Successful treatment with daratumumab for post-HSCT refractory hemolytic anemia.

Autoimmune cytopenias (AIC) following allogeneic hematopoietic stem cell transplantation (HSCT) may cause significant morbidity and mortality and are often challenging to treat. We present a case of a pediatric patient with primary myelofibrosis of infancy caused by VPS45 protein deficiency, who developed severe refractory hemolytic anemia and immune-mediated thrombocytopenia 3.5 months following HSCT. After the failure of several treatments, he received daratumumab, an anti-CD38 specific antibody, and demonstrated fast and sustained response. The only side effect was delayed recovery of humoral immunity. Daratumumab, by targeting antibody-producing plasma cells, may be a valid treatment option for refractory post-HSCT AIC.