Pediatric Hematology and Oncology

Studies on the long-term treatment outcomes of childhood acute lymphoblastic leukemia (ALL) in resource-limited countries are scarce. The purpose of this study was to assess the evolution of survival outcomes of pediatric ALL in a tertiary care center in Thailand over a 40-year period. We retrospectively reviewed the medical records of pediatric patients who were diagnosed with ALL and treated at our center between June 1979 and December 2019. We classified the patients into 4 study periods depending on the therapy protocol used to treat the patients (period 1: 1979-1986, period 2: 1987-2005, period 3: 2006-2013, and period 4: 2014-2019)...

This study investigates the prevalence of vitamin and iron deficiencies at cancer diagnosis. Newly diagnosed children between October 2018 and December 2020 at two South African pediatric oncology units (POUs) were assessed for nutritional and micronutrient status (Vit A, Vit B12, Vit D, folate, and iron). A structured interview with caregivers provided information regarding hunger and poverty risks. There were 261 patients enrolled with a median age of 5.5 years and a male-to-female ratio of 1:0.8. Nearly half had iron deficiency (47...

Malignant extracranial germ cell tumors (GCTs) are rare in pediatric patients and are usually extremely sensitive to chemotherapy. Relapsed or refractory tumors, although rare, established the need for second-line therapies, including high-dose chemotherapy with autologous stem cell transplantation (HDCT/ASCT). However, there are few data on its use in children with GCTs. We present a retrospective analysis of all patients diagnosed with extracranial GCTs who received HDCT/ASCT at two Brazilian pediatric cancer centers from May 1999 to December 2019...

Growth hormone deficiency (GHD) may occur in pediatric patients with central nervous system (CNS) tumors at initial tumor presentation or later as treatment-related sequelae. While it is well recognized that growth hormone (GH) has beneficial effects on growth and endocrinopathies, there's often hesitancy by clinicians to initiate GH therapy for GHD after CNS tumor diagnosis due to the perceived increased risk of tumor recurrence. The available data is described here and based on this review, there is no evidence of increased risk of tumor recurrence or secondary malignancy in patients treated with GH after CNS tumor diagnosis...

Prognostic predictive value of end of induction minimal residual disease (EOI-MRD) is well established in acute lymphoblastic leukemia (ALL). We evaluated the factors likely to affect EOI-MRD positivity (>0.01%) by flow cytometry and relapse in different BFM-95 (Berlin-Frankfurt-Munich) risk groups among children and adolescents. In this retrospective study, data of 223 newly diagnosed patients with ALL was analyzed. Association between demographic and pretreatment characteristics with EOI-MRD was assessed...

Pazopanib, a receptor tyrosine kinase inhibitor, exhibits anti-tumor activity in adult bone and soft-tissue sarcomas (STS), but has not yet been approved for pediatric tumors. The primary objective was to evaluate pazopanib efficacy when used alone or in combination with topotecan. This real-world multicenter retrospective study included patients with solid tumors, aged 25 years or less at the time of initial diagnosis, treated with pazopanib outside of a clinical trial. Nineteen patients were eligible for efficacy analysis: 14 bone tumors and 5 STS...

No abstract text is available yet for this article.

Osteonecrosis (ON) is a known complication of acute leukemia (AL) management, affecting 1%-10% of young patients and resulting in long-term morbidity. Widespread access to MRI over the past decade has allowed earlier detection and more accurate assessment. This study investigated clinical and MRI features of the 129 (2.5%) patients with symptomatic ON retrospectively recruited from the French LEA (Leucémies de l'Enfant et de l'Adolescent, or child and adolescent leukemias ) cohort ( n  = 4,973)...

Hereditary cancer predisposition accounts for more than 10% of all cancers in pediatric age group and this is increasingly recognized as an important entity because of modern sequencing techniques. We report a rare association of two concurrent cancer predisposition syndromes, BRCA2 and PMS2, in a young child who presented with concurrent malignancies including Wilms tumor, myelodysplastic syndrome and an indeterminate brain lesion who succumbed to his disease. Multiple synchronous malignancies present difficult clinical and psycho-social challenges which need to be carefully addressed in the setting of a multi-disciplinary team approach...

No abstract text is available yet for this article.

Introduction: Collaborative studies have contributed to improved survival of pediatric Hodgkin lymphoma in well-resourced settings, but few are documented in resource-constrained countries. The South Africa Children's Cancer Study Group initiated harmonization of management protocols in 2015. This article analyzes barriers and enablers of the process. Methods: Clinician-researchers at 11 state-funded pediatric oncology units completed preparatory questionnaires in June 2018. Parameters included infrastructure, access to therapeutic modalities and clinician numbers...

Patients with sickle cell disease (SCD) have a high risk for venous thromboembolism which is associated with increased risk of mortality. Studies examining risk of pulmonary embolism (PE) in children with SCD are lacking. This study was conducted in children with SCD between 0-21 years of age using a nationwide administrative database in the United States- Pediatric Health Information System (PHIS) from January 2010 to June 2021. Diagnostic codes and imaging, procedure, and pharmaceutical billing codes were used to identify PE and potential clinical, demographic, and utilization risk factors...

Neurofibromatosis Type 1 (NF1) is a neurocutaneous syndrome characterized by multiple café-au-lait macules, neurofibromas, and predisposition to malignancies, including rhabdomyosarcomas (RMS). Somatic NF1 mutations occur in RMS and other cancers, and ∼1% of patients with RMS have NF1. We describe three patients who presented prior to one year of age with RMS and were subsequently diagnosed with NF1. Compared to sporadic RMS, patients with this cancer predisposition syndrome are diagnosed younger, genitourinary sites are more common, and tumors are almost exclusively the embryonal subtype...

Patients with Langerhans cell histiocytosis (LCH) have been effectively treated with intravenous cytarabine. Intravenous or subcutaneous cytarabine infusions have been effective for leukemia patients, and pharmacokinetic studies have shown very similar blood levels of the drug with either route. We present three LCH patients treated with subcutaneous cytarabine either because intravenous access could not be maintained or due to patient refusal. One patient with pulmonary and skin LCH had a complete response...

Neuroblastoma (NB) is the most common extracranial solid tumor of childhood. The complete burden and outcomes in Uganda are unknown. The study was a multicenter retrospective chart review of children aged between 0 to 15 years diagnosed with NB from 2010 to 2020. Demographic, clinical and tumor-related characteristics were extracted for analysis. Kaplan-Meier survival curves and Cox regression models were used to determine the one-year overall survival (OS) and identify prognostic factors. Seventy-five patients were evaluated, with a median age at diagnosis of 48 months (IQR 26-108 months)...

A 13-year-old girl presented with hypoxemia during adjuvant chemotherapy for an osteosarcoma of the left distal femur. She underwent an amputation complicated by a post-operative pulmonary embolism (PE). Three months post-operatively, she was admitted to hospital with severe hypoxemia and diagnosed with pulmonary hypertension on echocardiogram in the context of extensive bilateral PE on computed tomography. She was planned for elective pulmonary thromboendarterectomy, but rapidly deteriorated requiring emergent surgery...

The presence of an anterior mediastinal mass should prompt rapid triage, workup and treatment to effectively manage and prevent emergent complications. Implementation of an AMM protocol can ensure the response is standardized and coordinated. Importantly, such a protocol can encourage prompt multi-disciplinary communication to mitigate risks associated with procedures required for timely diagnosis. The aim of this review is to evaluate the BC Children's Hospital's Pediatric New/Suspected Anterior Mediastinal Mass (AMM) Protocol...

Indonesia is a rapidly growing lower-middle-income country (LMIC) located in Southeast Asia. It has 267.3 million inhabitants, with 31.6% (84.4 million) children. According to GLOBOCAN 2020, Indonesia had the highest prevalence of pediatric cancer cases in Southeast Asia (43.5%), and brain tumors had the third-highest incidence in Indonesia. Treating children with brain tumors with radiotherapy is challenging, especially the late treatment effects that can affect their quality of life (QoL). This study aimed to show the QoL in children with brain tumors after radiotherapy in Dr...

No abstract text is available yet for this article.

Strokes affect up to 10% of children with sickle-cell disease (SCD). The most commonly used strategy to prevent a first-time stroke or its recurrence is to perform periodic red blood cell transfusions. This article aims to evaluate the quality of life (QoL) of children and adolescents with SCD undergoing a chronic transfusion regimen (CTR) for stroke prophylaxis, according to their caregivers' perception. A cross-sectional study was conducted using a sociodemographic interview with an application of a validated instrument (Pediatric Quality of Life Inventory) involving 16 caregivers of patients with SCD aged <18 years undergoing CTR in a reference center...