[The rise of targeted therapies in pediatric oncology]. / L'essor des thérapies ciblées en oncologie pédiatrique.

Cancers are rare pathologies in children. Improvement in survival rates has been obtained thanks to new therapeutic strategies based on the identification of risk factors. Targeted therapies in paediatric oncology are new treatments providing hope that cure is achievable without long-term sequelae.

Les cancers pédiatriques sont des pathologies rares. L'amélioration du taux de survie a été obtenue par de nouvelles stratégies de traitement basées sur l'identification de facteurs de risque. Les thérapies ciblées en oncologie pédiatrique, nouvelle arme thérapeutique, sont porteuses d'espoir de guérison, sans séquelles à long terme.

[What follow-up after pediatric cancer ? The SALTO consultation experience]. / Quel suivi après un cancer pédiatrique ? L'expérience de la consultation SALTO.

During the past 50 years, the mortality due to childhood cancers decreased dramatically thanks to improvements in supportive care and the use of multimodal approaches. In this context, the long-term follow up after childhood cancer has become a main concern for pediatric oncologists. The SALTO programme was developed in 2012 at the CHR Citadelle in Liège in order to organize a multidisciplinary long-term follow-up for the patients previously treated in our department for a childhood cancer. The aim of the present study was to review, for the most frequent childhood cancers, the long-term sequellae and the second cancers developed by the patients participating to the SALTO programme in order to define the follow-up needed. Our data confirm the importance of a multidisciplinary long-term follow-up, based on the treatments received and following international guidelines.

Au cours des cinquante dernières années, la mortalité liée aux cancers pédiatriques a fortement diminué grâce à une amélioration des soins de support et à l'utilisation d'approches multimodales. Dans ce contexte, le devenir à long terme des patients guéris d'un cancer pédiatrique est devenu une des préoccupations majeures pour les oncologues pédiatres. Dans cette optique, la consultation SALTO («Suivi À Long Terme en Oncologie¼) a été mise en place en 2012 au CHR de la Citadelle pour assurer le suivi multidisciplinaire des adultes ayant été traités dans notre secteur d'hémato-oncologie pédiatrique. L'objectif de cette étude a été de revoir, pour les cancers pédiatriques les plus fréquents, les séquelles et les tumeurs secondaires présentées par les patients suivis en consultation SALTO afin de préciser les modalités du suivi au long cours après cancer pédiatrique. Nos résultats confirment l'importance d'un suivi multidisciplinaire à long terme adapté aux traitements reçus, sur base de recommandations internationales.

[Coexistence of sickle cell disease and systemic lupus erythematosus]. / Co-existence d'une drépanocytose et d'un lupus érythémateux disséminé.

Sickle cell disease and systemic lupus erythematosus (SLE) are two distinct chronic conditions sharing many clinical manifestations. Coexisting of these two diseases is rare, but it is important to make the diagnosis of SLE in sickle cell patients to initiate appropriate treatment and limit the risk of complications. High titers of autoantibodies have been reported in sickle cell patients as well as a higher risk of immune deficiency. These patients present a defective activation of the alternative pathway of the complement that increases the risk of infection with encapsulated bacteria and a difficulty in eliminating antigenes that could predispose to autoimmune diseases. We report three case of children with sickle cell disease. They developed symptoms initially attributed to sickle cell disease, but after investigations revealed an underlying SLE.

La drépanocytose et le lupus érythémateux disséminé (LED) sont deux pathologies chroniques distinctes partageant certaines manifestations cliniques communes. La coexistence de ces deux pathologies est rare. Cependant, il est important de faire le diagnostic de LED chez ces patients drépanocytaires afin de pouvoir instaurer un traitement adéquat et limiter le risque de complications. Des hauts titres d'anticorps ont été décrits chez les patients drépanocytaires ainsi qu'un risque plus élevé de déficits immunitaires. Ces patients présentent un défaut dans l'activation de la voie alterne du complément qui augmente le risque d'infection à bactéries encapsulées et un défaut d'élimination d'antigènes qui pourrait prédisposer aux maladies auto-immunes. Nous décrivons le cas de trois patientes drépanocytaires majeures. Elles ont développé des symptômes attribués initialement à la drépanocytose, mais qui se sont par la suite révélés être un LED.

[Epidemiology of childhood cancer, a single-center study (1985-2016)]. / Epidémiologie des cancers de l'enfant, une étude monocentrique (1985-2016).

Cancer is the second leading cause of death among children aged 5 to 14, after accidents. We conducted a study on the epidemiology of childhood cancer in the university pediatric oncology department of the CHU-CHR in Liège, Belgium. We studied a cohort of 662 patients between the ages of 0 and 17 whose malignancy diagnosis was made between 1985 and 2016. The analyzes were performed retrospectively using medical files. The number of new cases, the proportion of different cancers, sex ratio, age at diagnosis and survival at 5 and 10 years were the epidemiological factors studied.We have been able to show an increase in the number of new diagnoses per year. More than 40 % of childhood cancers occur before the age of five. The most common neoplasias are leukemias, tumors of the central nervous system and lymphomas. This distribution is influenced by age. All malignant tumours combined, we observed a slightly larger proportion of affected boys than girls. Overall survival at 5 years reaches 80.2 %. However, it varies according to the type of tumour from 59.3 % for malignant soft tissue tumors up to 100 % for hepatoblastomas.

Le cancer est la deuxième cause de décès chez les enfants de 5 à 14 ans, après les accidents. Nous avons réalisé une étude sur l'épidémiologie des cancers de l'enfant au sein du service universitaire d'oncologie pédiatrique du CHU-CHR de Liège. Nous avons étudié une cohorte de 662 patients, âgés de 0 à 17 ans, dont le diagnostic de tumeur maligne a été posé entre 1985 et 2016. Le nombre de nouveaux cas, la proportion des différents cancers, le sex ratio, l'âge au diagnostic et la survie à 5 et 10 ans ont été les facteurs épidémiologiques étudiés. Nous avons pu démontrer une augmentation du nombre de nouveaux diagnostics par an. Plus de 40 % des cancers de l'enfant surviennent avant l'âge de 5 ans. Les néoplasies les plus fréquentes sont les leucémies, les tumeurs du système nerveux central et les lymphomes. Cette répartition est néanmoins influencée par l'âge. Toutes tumeurs malignes confondues, nous avons observé une proportion légèrement plus grande de garçons atteints que de filles. La survie globale à 5 ans s'élève à 80,2 %. Elle varie cependant selon le type de tumeur de 59,3 % pour les tumeurs malignes des tissus mous jusqu'à 100 % pour les hépatoblastomes.

Screening protocols to monitor respiratory status in primary immunodeficiency disease: findings from a European survey and subclinical infection working group.

Many patients with primary immunodeficiency (PID) who have antibody deficiency develop progressive lung disease due to underlying subclinical infection and inflammation. To understand how these patients are monitored we conducted a retrospective survey based on patient records of 13 PID centres across Europe, regarding the care of 1061 adult and 178 paediatric patients with PID on immunoglobulin (Ig) G replacement. The most common diagnosis was common variable immunodeficiency in adults (75%) and hypogammaglobulinaemia in children (39%). The frequency of clinic visits varied both within and between centres: every 1-12 months for adult patients and every 3-6 months for paediatric patients. Patients diagnosed with lung diseases were more likely to receive pharmaceutical therapies and received a wider range of therapies than patients without lung disease. Variation existed between centres in the frequency with which some clinical and laboratory monitoring tests are performed, including exercise tests, laboratory testing for IgG subclass levels and specific antibodies, and lung function tests such as spirometry. Some tests were carried out more frequently in adults than in children, probably due to difficulties conducting these tests in younger children. The percentage of patients seen regularly by a chest physician, or who had microbiology tests performed following chest and sinus exacerbations, also varied widely between centres. Our survey revealed a great deal of variation across Europe in how frequently patients with PID visit the clinic and how frequently some monitoring tests are carried out. These results highlight the urgent need for consensus guidelines on how to monitor lung complications in PID patients.

Paravertebral Burkitt's Lymphoma in a Child: An Unusual Presentation.

Paravertebral malignant tumors constitute 4.8% of cancer cases in pediatric oncology and are mostly composed of neuroblastoma (46.4%) and soft tissue sarcomas (35.7%). We describe the case of a Caucasian 6-year-old boy who was admitted for middle back pain radiated to limbs and progressively increasing weakness of the legs, suggesting a spinal cord disease. The exploration revealed two paravertebral masses extending through the neural foraminae into the epidural space. The association with elevated serum neuron specific enolase suggested at first the diagnosis of neuroblastoma, but the pathological examination revealed a Burkitt's lymphoma. This is a rare location of sporadic Burkitt's lymphoma with neurologic syndrome as first symptoms.

[Borrelia-associated lymphocytoma cutis]. / Le lymphocytome borrélien du lobe de l'oreille.

We describe a 6-year-old boy who developed Borrelia burgdorferi-associated lymphocytoma cutis on the ear. Lymphocytoma is a benign polyclonal B-cell lymphoproliferative process; it is defined as a subacute manifestation of early disseminated borrelial infection. Clinical history, physical examination, and serodiagnosis tests are often sufficient to establish diagnosis, but sometimes, histopathologic analysis is needed to exclude malignant cutaneous lymphomas. The outcome is always favorable but after antibiotic therapy, the lesion disappears promptly.

[Renal tumors in children. A single center study of 31 cases]. / Les tumeurs rénales de l'enfant. Une etude monocentrique: a propos de 31 cas.

In order to illustrate epidemiologic features and survival rate, 31 Wilm's tumours treated in our institution have been retrospectively studied. The mean age at diagnosis in our series was surprisingly higher than usually described: 25% of the patients were older than 8 years. Moreover, a mesoblastic nephroma congenital kidney tumour--appeared in a 10 month old girl. Symptoms were usually abdominal mass or gross hematuria. Young children are often brought to medical attention because of abdominal swelling: a large flank mass was palpable in all children under 5 years. Our study shows that preoperative chemotherapy results in a less intensive treatment regimen conducting probably to less sequellae. Event free survival and overall survival were respectively 87% and 94% at 10 years after diagnosis. Renal insufficiency was the most important side effect: 2 children required renal transplantation.

Increased expression of bone sialoprotein in bone metastases compared with visceral metastases in human breast and prostate cancers.

The recent demonstration that bone sialoprotein (BSP) is expressed in osteotropic cancers suggests that this bone matrix protein might be implicated in the preferential seed and growth of metastatic cells in bone. High expression of BSP in breast and prostate primary carcinomas is associated with progression and bone metastases development. The exact mechanisms by which BSP may favor bone metastases formation are not clearly established yet. Although BSP expression has been detected in breast, prostate, lung, thyroid, and neuroblastoma primary tumors, no information regarding its expression in metastases is available to date. In this study, we have examined BSP expression in 15 bone and 39 visceral metastatic lesions harvested from 8 breast cancer patients and 7 prostate cancer patients who died of disseminated disease. We were able to retrieve the primary lesions from 5 of the 8 breast cancer patients as well as from all 7 prostate cancer patients. All the primary breast tumor patients and 5 of the 7 primary prostate cancer patients expressed a detectable level of BSP. Bone metastases from all 8 breast cancer patients and from 5 out of 7 prostate cancer patients exhibited detectable levels of the protein. Metastatic cells in close contact with bone trabeculae usually were highly positive for BSP. BSP also was detected in secondary lesions developed at visceral sites including liver, thyroid, lung, and adrenal glands. However, BSP expression was significantly lower in visceral metastases than in skeletal ones (Mann-Whitney test, p < 0.05). Our data represent the first demonstration of an increased expression of BSP in bone metastases compared with nonskeletal metastases in human breast and prostate cancers and add weight to the body of evidence attributing a significant role to this protein in the genesis of bone metastases.