Mixed phenotype acute leukemia (MPAL) is a relatively rare and heterogeneous group of diseases, which account for 2-5% of leukemia. So far, no optimal treatment regimens have been established for MPAL patients. CD19 chimeric antigen receptor T (CAR T) therapy have shown remarkable efficacy in B-ALL patients. In this study, 5 MPAL patients received CD19 CAR T cell infusion and 3 out of 5 patients (60%) achieved complete response (CR). 2 of 5 patients had only mild cytokine release syndrome (CRS). Neurotoxicity was not observed. Our data indicate that CD19 CAR T therapy is safe and effective for majority refractory/relapsed (r/r) MPAL patients.
Letter to the Editor: Interesting clinical observationTitle: Acute Exacerbation of Graft-versus-Host Disease following SARS-CoV2 infection after Hematopoietic Stem Cell Transplant in Two Pediatric PatientsRunning Title: Graft-versus-Host Disease and SARS-CoV2 infectionAuthors: Michelle Choe1,2* Caridad Martinez1,2, Robert Krance1,2, Erin Doherty1,2ORCID:Michelle Choe: 0000-0001-9850-9435Institutional Affiliation:1 Hematology and Cancer Center, Texas Children’s Hospital, Houston, Texas2 Department of Pediatrics, Baylor College of Medicine, Houston, Texas*Corresponding Author:Michelle Choe, MD6701 Fannin St. Ste. 1510Houston, TX 77030P: 832-822-4242F: 832-825-1453Abbreviations key:
Introduction: Hematologic complications of SARS-CoV-2 infection are well described in hospitalized adults with correlation to adverse outcomes. Information published in children has been limited. Methods: An international multi-centered retrospective registry was established to collect data on the clinical manifestations of SARS-CoV-2 or multisystem inflammatory syndrome (MIS-C) in hospitalized children between February 1, 2020 – May 31, 2021. This sub-study focused on hematologic manifestations. Study variables included patient demographics, comorbidities, clinical presentation, course, laboratory parameters, management, and outcomes. Results: Nine hundred and eighty-five children were enrolled and 915 (93%) had clinical information available; 385 (42%) had symptomatic SARS-CoV-2 infection upon admission, 288 had MIS-C (31.4%) and 242 (26.4%) had alternate diagnosis with SARS-CoV-2 identified incidentally. During hospitalization, 10 children (1%) experienced a thrombotic event, 16 (1.7%) had hemorrhage and 2 (0.2%) had both thrombotic and hemorrhagic episodes. Significant prothrombotic comorbidities included congenital heart disease (p-value = 0.007), central venous catheter (p = 0.04) in children with primary SARS-CoV-2 infection; and obesity (p-value= 0.002), cytokine storm (p= 0.012) in those with MIS-C. Significant pro- hemorrhagic conditions included age > 10 years (p = 0.04), CVC (p= 0.03) in children with primary SARS-CoV-2infection; and thrombocytopenia (0.001), cytokine storm (0.02) in those with MIS-C. Eleven patients died (1.2 %) with no deaths attributed to thrombosis or hemorrhage Conclusion: Thrombotic and hemorrhagic complications are uncommon in children with SARS-CoV-2 infection and observed with underlying co-morbid conditions. Understanding the complete spectrum of hematologic complications in children with SARS-CoV-2 infection or MIS-C requires ongoing multi-center studies.
Youth with sickle cell disease (SCD) and their caregivers are susceptible to stress and depression, perhaps exacerbated by pandemic-associated health and economic concerns. Most of the 50 youth-caregiver dyads enrolled in the multi-site HABIT trial took an on-line survey of self-reported mental health symptoms and food insecurity during the 2020 COVID-19 pandemic. Compared to largely pre-pandemic results, prevalence of mental health symptoms in dyad members appeared to have shifted: fewer youth and more caregivers were affected during the pandemic; many of both groups lacked optimism. Pandemic screening of youth with SCD for mental health symptoms and food insecurity appears warranted.
Because they can experience neutropenia due to bone marrow failure, patients with Shwachman-Diamond syndrome (SDS) carry increased risk for serious infections compared to the general population; however, it has been unknown whether this predisposes them to COVID-19 infection or more significant complications. We compiled results from a survey distributed to participants in the Shwachman-Diamond Syndrome Registry between May and June 2021. In this report we describe the characteristics and outcomes of patients with SDS who had COVID-19. Patients reported a short clinical course without significant complications or severe cytopenias. Additionally, COVID-19 vaccines were well tolerated with only minor side effects.
Background It is unclear how intensity-modulated radiation therapy (IMRT) impacts long-term risk of second malignant neoplasms (SMNs) in childhood cancer patients. Procedure Patients aged 10 years, many solid SMNs after IMRT in childhood cancer survivors develop in the high dose region. These data serve as a foundation for comparison with other modalities of radiation treatment (e.g., proton therapy).
Objective: To synthesise existing qualitative research exploring the experiences of parents caring for children with cancer during the end-of-life phase, and the factors that influence parental decision making when choosing the location of end-of-life care and death for their child. Results: This review included 15 studies involving 460 parents of 333 children and adolescents who died from progressive cancer. Where reported, the majority (58%) of children died at home or in a hospital (39%), with only a small fraction dying in a hospice. Factors impacting decision-making for location of care included the quality of communication and the quality of care available. Themes related to choosing home for end-of-life care and death included: honouring the child’s wishes, familiarity of home, and parents’ desire to be their child’s primary carer. Preference for location of death in hospital included trust in hospital staff, practical logistics and the safety of the hospital environment.
We report the first case series of 14 children with intracranial germ cell tumour (iGCT) and concomitant central diabetes insipidus (DI), who developed hyponatremia secondary to renal salt wasting syndrome (RSWS) following the administration of carboplatin. Clinicians prescribing platinum-based chemotherapy for this group of patients should be alert to the risk of RSWS. Regular monitoring should be performed as hyponatraemia can be asymptomatic until it is severe.
Tristan Boam1, Bethan G Rogoyski2, Melissa Gabriel3, Paul D Losty4Department of Paediatric Surgery, Leicester Royal Infirmary, Leicester, UKLeicester School of Allied Health Sciences, De Montfort University, Leicester, UKDepartment of Urology, Norfolk and Norwich Hospital, Norwich, UKAlder Hey Children’s Hospital NHS Foundation Trust, School of Health and Life Science, University of Liverpool, UK
Pheochromocytoma and paraganglioma (PPGL) are rare neuroendocrine tumors in childhood. Cancer predisposition syndromes (CPS) are increasingly recognized as the underlying cause for a number of pediatric malignancies and up to 40% of PPGL are currently thought to be associated with a hereditary predisposition1,2. With the increasingly widespread availability of functional molecular imaging techniques, nuclear medicine imaging modalities such as 18F-FDG-PET/CT, 123I-MIBG SPECT/CT, and 68Ga-DOTATATE PET/CT now play an essential role in the staging, response assessment and determination of suitability for targeted radiotherapy in patients with PPGL. Each of these imaging modalities targets a different cellular characteristic, such as glucose metabolism (FDG), norepinephrine transporter expression (MIBG), or somatostatin receptor expression (DOTATATE), and therefore can be complementary to anatomic imaging and to each other. Given the recent FDA approval3 and increasing use of 68Ga-DOTATATE for imaging in children4, the purpose of this article is to use a case-based approach to highlight both the advantages and limitations of DOTATATE imaging as it compares to current radiologic imaging techniques in the staging and response assessment of pediatric PPGL, and to offer a decision algorithm for the use of functional imaging that can be applied to PPGL, as well as other neuroendocrine malignancies.
BACKGROUND/OBJECTIVES: Survivors of pediatric brain tumors (BT) are at increased risk for difficulties with social competence, including poor social information processing (SIP) and peer relationships. Due to improved survival rates among BT, there is a need to better understand these challenges and if they are specific to BT versus other survivors of childhood cancer. METHODS: 51 BT and 34 survivors of pediatric solid tumors (ST) completed evaluations of SIP and peer relationship quality within 6 months of completing treatment and at one year follow-up. Caregivers also completed a measure of social skills. Linear mixed models evaluated (1) differences between BT and ST on SIP and social skills and (2) how indices of SIP were associated with peer relationships over time for ST and BT. RESULTS: BT did not differ from ST on indices of SIP or social skills over time. There was a three-way interaction between measures of SIP, group, and time to predict peer relationships. ST showed a positive association between baseline social skills and theory of mind and peer relationships over time, whereas BT showed an inverse association between baseline social skills and theory of mind and peer relationships over time. CONCLUSION: Baseline SIP and social skills affected the trajectory of BT peer relationships. BT social functioning should be monitored regularly after the completion of treatment to determine if and when intervention services would be beneficial.
The mRNA COVID-19 vaccine and COVID-19 infection caused by the SARS-CoV-2 virus may be immunologic triggers for the development of thrombotic thrombocytopenic purpura (TTP). There is not yet literature that discusses TTP induced by COVID-19 vaccination or infection in pediatric or adolescent patients. We describe 4 adolescents presenting with TTP (both de novo and relapsed disease) following administration of the Pfizer COVID-19 vaccine or after COVID-19 infection. Our observations demonstrate that the Pfizer-BioNTech mRNA vaccine and COVID-19 infection can act as triggers for the development/relapse of both congenital and acquired TTP.
While treatment protocols for Hodgkin Lymphoma (HL) are well established, there is no literature available to guide therapy or estimate prognosis for patients with Fontan physiology who develop HL. The physiology of a Fontan procedure can result in the inability to tolerate chemotherapy toxicities, supportive care and infection. We present a series of 3 patients with Fontan physiology who were treated for HL and discuss their clinical course and treatment.
Supraclavicular Lymphadenopathy after COVID-19 vaccinationKelsey Larkin 1, Archana Sharma DO2, Richard Drachtman MD,2, Gratian Salaru , MD31 Robert Wood Johnson Medical School2Division of Pediatric Hematology, Rutgers Cancer Institute, Robert Wood Johnson Medical School3Department of Pathology, Robert Wood Johnson Medical SchoolCorrespondence to Archana Sharma, DO, Rutgers Cancer Institute of NJ, 195 Little Albany Street, Room 3507, New Brunswick, NJ 08901.Telephone: 732-235-8864Sharmaar@cinj.rutgers.eduText Word Count: 974Brief running title: Lymphadenopathy after COVID-19 vaccination.Key words: lymphadenopathy, COVID-19, vaccine,Images: 2