[Haematopoietic stem cell donation from minor donor: Respecting laws, assessing fitness, delivering information and good care (SFGM-TC)]. / Don de cellules souches hématopoïétiques à partir d'un donneur mineur : cadre réglementaire, information, évaluation de l'aptitude au don et parcours (SFGM-TC).

Haematopoietic stem cell collection from paediatric donors is a common and life-saving practice, as evidenced by the fact that there is a growing annual number of cases of transplants from minor donors among SFGM-TC centers over the last decade. Still, medical use of human tissue from a healthy and underage donor requires proper regulations and medical management. The guidelines below aim at underlining the importance of pondering the legal, medical and ethical aspects of using stem cells from healthy paediatric donors and stress out the importance of obtaining informed consent at the time of assessing HLA compatibility. Combined medical and psychological assessments are required before the donation, as well as one month later and one year later to ensure of the child's physical and mental wellbeing. Bone marrow harvest under general anaesthetics remains the preferred method of collection for children. Peripheral blood stem cell collection should only be considered for children who will not require a central venous access for collection. We aim at offering guidelines centered on the healthy child donating stem cells and his/her wellbeing, and these should be regularly reviewed as medical practices evolve.

Upper and/or Lower Respiratory Tract Infection Caused by Human Metapneumovirus After Allogeneic Hematopoietic Stem Cell Transplantation.

BACKGROUND: Human metapneumovirus (hMPV) epidemiology, clinical characteristics and risk factors for poor outcome after allogeneic stem cell transplantation (allo-HCT) remain a poorly investigated area. METHODS: This retrospective multicenter cohort study examined the epidemiology, clinical characteristics, and risk factors for poor outcomes associated with human metapneumovirus (hMPV) infections in recipients of allo-HCT. RESULTS: We included 428 allo-HCT recipients who developed 438 hMPV infection episodes between January 2012 and January 2019. Most recipients were adults (93%). hMPV infections were diagnosed at a median of 373 days after allo-HCT. The infections were categorized as upper respiratory tract disease (URTD) or lower respiratory tract disease (LRTD), with 60% and 40% of cases, respectively. Patients with hMPV LRTD experienced the infection earlier in the transplant course and had higher rates of lymphopenia, neutropenia, corticosteroid use, and ribavirin therapy. Multivariate analysis identified lymphopenia and corticosteroid use (>30 mg/d) as independent risk factors for LRTD occurrence. The overall mortality at day 30 after hMPV detection was 2% for URTD, 12% for possible LRTD, and 21% for proven LRTD. Lymphopenia was the only independent risk factor associated with day 30 mortality in LRTD cases. CONCLUSIONS: These findings highlight the significance of lymphopenia and corticosteroid use in the development and severity of hMPV infections after allo-HCT, with lymphopenia being a predictor of higher mortality in LRTD cases.

[Pediatric refractory acute immune thrombocytopenic purpura secondary to asymptomatic SARS-CoV2]. / Face à la COVID-19. Purpura thrombopénique immunologique pédiatrique réfractaire secondaire à une infection asymptomatique à SARS-CoV2.

Acute immune thrombocytopenic purpura (ITP) has been revealed as an uncommon complication of COVID-19 in children. Severe bleeding may occur but is rarely life threatening. Management is based on the severity of bleeding symptoms and the degree of thrombocytopenia. We report the case of a 7-year-old girl with severe acute ITP secondary to a COVID-19 infection -without any respiratory symptoms. The initial clinical examination showed a large bulging mediodorsal hematoma, purpuric lesions, and posterior pharyngeal hemorrhage. The patient was monitored in a pediatric intensive care unit. Initial medical management consisted of intravenous immunoglobulins and systemic steroids. Despite this treatment, bleeding and thrombocytopenia worsened, and secondary macroscopic haematuria occurred, requiring 6-hourly platelet transfusions and increased steroid doses to obtain sufficient hemostasis. This case presents a rare and severe acute pediatric ITP secondary to asymptomatic SARS-COV2 which was refractory to initial management and opens the discussion to second line therapeutic interventions.

Le purpura thrombopénique immunologique aigu (PTI) s'est révélé comme une complication inhabituelle de la COVID-19 en pédiatrie. Une hémorragie sévère peut survenir, mais constitue rarement une menace vitale. La prise en charge dépend de la sévérité des signes hémorragiques et du niveau de la thrombopénie. Nous rapportons le cas clinique d'une enfant de 7 ans avec diagnostic de PTI sévère aigu secondaire à la COVID-19, sans symptôme respiratoire. L'examen clinique initial mettait en évidence un large hématome médiodorsal bombant, des lésions purpuriques, ainsi qu'un saignement pharyngé postérieur. Une surveillance en unité de soins intensifs avec administration d'immunoglobulines intraveineuses et de corticoïdes systémiques a été initiée. Malgré la thérapeutique, les saignements se sont intensifiés, avec apparition secondaire d'une hématurie macroscopique justifiant la réalisation de transfusions plaquettaires continues et la majoration des doses de corticoïdes jusqu'à l'obtention de l'hémostase. Ce cas clinique relate un cas rare et sévère de PTI aigu pédiatrique secondaire à une infection à SARS-COV2 réfractaire au traitement habituel et ouvre la discussion aux thérapeutiques de deuxième ligne.

Efficacy of Cytomegalovirus Specific Immunoglobulins to Reduce CMV Reactivation in Pediatric Hematopoietic Stem Cell Transplant Recipients.

Cytomegalovirus (CMV) infection is a serious complication of pediatric hematopoietic stem cell transplant (HSCT). To date, antiviral therapy has been the mainstay of prophylaxis, with conflicting results regarding the benefits of CMV-specific immunoglobulins (CMV-Ig). After introducing prophylactic CMV-Ig to HSCT recipients at risk (seropositive recipient and/or donor), we conducted a single-center retrospective study comparing the incidence and severity of CMV infection with and without CMV-Ig. We identified 49 'at risk' recipients from 76 consecutive HSCTs over 3.5 years, in addition to standard antiviral prophylaxis, 10 patients received CMV-Ig and 39 did not. There was no significant difference in donor type, cell source, conditioning, or CMV status between the groups. We observed a potential trend toward reduction of incidence of CMV reactivation in patients exposed to CMV-Ig (30%) compared with those who weren't (38.4%). Besides, no symptomatic or lethal infection was observed in the CMV-Ig group, and time to recovery seemed shorter (21 [±7] vs 51.4 [±55] days) and peak titers lower (4578 [±4788] vs 24131 [±49257]) with CMV-Ig. No adverse events were noted. The statistical significance of the results was limited by the small sample size. These data raise interest in prophylactic CMV-Ig as a safe way of potentially reducing the severity and duration of CMV reactivation in HSCT.

Optic Nerve Germinoma and Transient Spontaneous Regression-More Than Meets the Eye.

Infiltration of the optic pathway by germ cell tumors is exceptional and can lead to confusion with glioma or inflammatory conditions. We present the case of a 14-year-old girl with an optic nerve germinoma extending to the hypothalamus and manifesting as panhypopituitarism and visual loss. The patient experienced spontaneous regression of the lesion followed by secondary deterioration requiring treatment. Four other cases of spontaneously regressing intracranial germinoma followed by regrowth have been reported in the literature. This report highlights the importance of clinical and radiologic monitoring of intracranial germinoma, even in the event of initial spontaneous improvement.

Methotrexate-induced Acute Myelopathy in a Teenager With High-risk Acute Lymphoblastic Leukemia.

Acute lymphoblastic leukemia (ALL) is one of the most frequent malignancies in childhood whose long-term survival has increased up to 80% thanks to modern therapy enhancements. Nevertheless, methotrexate (MTX) remains a mainstay of ALL therapy, but also represents one of the major causes of neurotoxicity in patients with ALL. MTX-induced toxicity occurs in about 9% of patients treated for ALL. It usually affects deep white matter region leading to leukoencephalopathy, which has varying clinical manifestations ranging from acute neurologic disturbances to seizures or chronic permanent encephalopathy. Here we describe a 13-year-old girl affected with ALL who developed lower limbs hypesthesia and static ataxia due to transverse myelopathy after intrathec administration of MTX therapy. A high-dose corticotherapy combined to vitamin supplementation and rehabilitation was tested. Neurological evolution was characterized by slow and partial recovery.

Neonatal Cancer Epidemiology and Outcome: A Retrospective Study.

OBJECTIVE: Our study aimed at describing neonatal cancer incidence, distribution by type, location, outcome, and long-term toxicity, by comparison with tumors occurring later in infancy. METHODS: The authors led a single-center retrospective analysis of 118 cases of tumors diagnosed in the first year of life and compared tumors' types incidence, presentation, location, and outcome according to age group at diagnosis (below or over 28 d of life). RESULTS: The most frequent neonatal tumor types in our series were germ cell tumors, mainly teratoma, followed by neuroblastoma and renal tumors, whereas in children below 1 year of age, brain tumors, neuroblastoma, and leukemia were the most common types. Genetic predisposition syndromes were present in 14% of these infants and antenatal sonography enabled 68% of diagnosis for tumors presenting at birth. Other patients presented with mass syndrome, hydrops, or skin lesions. Six percent of neonates with cancer died from their malignancies, and up to 18% experienced a chronic health condition as a consequence of therapy. CONCLUSIONS: Tumor pattern differs in neonates and infants, with a higher percentage of benign tumors in children below 28 days of life. Yet, long-term therapy-related toxicity is significant in younger patients. Enhancing knowledge of neonatal tumors, their epidemiology, clinical presentation, genetic background, and prognosis should help promote better management and introduce follow-up programs to improve surviving rates and the quality of life of survivors.

Endocrine consequences of neuroblastoma treatment in children: 20 years' experience of a single center.

Background Neuroblastoma (NBL) is a child neoplasia affecting extracranial tissue of neuroectodermal origin. It accounts for 10% of solid malignancies in children and is characterized by a survival rate approaching 70%, confronting physicians with the emergence of an adult survivor population who have been previously exposed to surgery, cytotoxic drugs, radiation therapy or metaiodobenzylguanidine (MIBG) therapy. All these treatments potentially affect the endocrine system. Our study consists in a retrospective review of late endocrine effects arising in survivors treated for NBL during childhood. Methods The medical files of 47 patients (M/F = 26/21) treated for NBL were reviewed. Collected data consisted of age, height, weight and biological hormonal values at diagnosis and at the last follow-up consultation. The incidence of late effects in our sample was compared to the data from the literature. Results Patients were between 0 and 15.8 years of age at diagnosis (median: 1.16 years) and between 1 and 25 years of age at last follow-up (median: 16 years). Twenty-six patients were treated with chemotherapy (CT), 11 underwent CT and radiation therapy and five were treated with CT and MIBG therapy. Ten percent of the patients died before reaching the end of therapy. Late effects occurred in 54% of the patients. Thirty-six percent of patients had non-endocrine complications (musculoskeletal, neurological, hematological or hepatic chronic conditions). Endocrine complications (28%) affected mainly patients treated with CT and consisted of gonadal dysfunction (up to 42% patients of over 12 years of age at follow-up) and hypothyroidism (21%). Our analysis revealed that CT had a significant impact on final height (p < 0.05). Conclusions Treatment for childhood malignancies exposes children to late effects affecting the endocrine system. In children treated for NBL, hypothyroidism, gonadal failure and impaired growth appear to be the main endocrine complications. Close follow-up of survivors is thus appropriate.

Studying self-awareness in children: validation of the Questionnaire of Executive Functioning (QEF).

OBJECTIVE: People with accurate representations of their own cognitive functioning (i.e. cognitive self-awareness) tend to use appropriate strategies to regulate their behavior. Due to the lack of appropriate instruments, few studies have examined the development of this ability among children. METHOD: This study tested the measurement properties of the self-rating and other-rating forms of the Questionnaire of Executive Functioning (QEF), designed to tap children's knowledge of their executive functioning. Specifically, the construct, convergent, and discriminant validities were investigated and a self-other discrepancy score was computed to assess children's executive self-awareness. Participants were 317 children aged 7-14 years old. RESULTS: Confirmatory factor analyses carried out on the QEF confirmed the eight-factor structure of both versions. There were significant correlations between the QEF and the parent versions of the Behavior Rating Inventory of Executive Function, the Dysexecutive Questionnaire for Children, and the Childhood Executive Functioning Inventory. Both forms of the QEF were able to distinguish between children who had sustained a traumatic brain injury (TBI) and control participants. A statistical difference was observed between the TBI and control groups on this score, suggesting that TBI may trigger self-awareness impairments in children. CONCLUSION: The good psychometric properties of the two forms of the QEF were established. Furthermore, results of the analyses carried out on the different discrepancy scores seem to indicate that the QEF could help clinicians to detect patients with self-awareness deficits.

Concomitant nodal involvement by Langerhans cell histiocytosis and Hodgkin's lymphoma.

A 10-year-old girl with a family history of Hodgkin's lymphoma presented with a 2 month history of cervical lymphadenopathy and weight loss. Biopsy indicated concomitant nodal involvement by Langerhans cell histiocytosis and Hodgkin's lymphoma. Such an association is rare, especially so in children, but is not an isolated phenomenon, thereby prompting the question of whether Langerhans cell histiocytosis is a reactive or a neoplastic process.