Verena Wiegering

• Medical Doctor at University of Wuerzburg

Background: The management of rare tumors at reference centers with specialized multidisciplinary tumor boards (MTBs) improves patient survival. No international standardized diagnostic and treatment recommendations for pediatric patients with adrenocortical carcinoma (ACC) exist so far. The aim of this case-based study is to determine how congruen...

Inflammation-based scores have been demonstrated to be independent prognostic factors in predicting outcomes in adult ACC. We aimed to investigate the prognostic role of these scores in pACC patients. An international, multicenter analysis was conducted on a pediatric cohort from 21 ACC centers. Pretreatment inflammation-based scoring parameters, i...

Therapeutic options of advanced pediatric adrenocortical carcinoma (pACC) are limited, and achieving valuable risk stratification remains challenging. We refined the value of prognostic factors, with an emphasis on resection status. Retrospective, international data from 106 patients with advanced pACC from various collaborating centers of the inte...

Objective Mitotane is an important cornerstone in the treatment of pediatric adrenal cortical tumors (pACC), but experience with the drug in the pediatric age group is still limited and current practice is not guided by robust evidence. Therefore, we have compiled international consensus statements from pACC experts on mitotane indications, therapy...

BACKGROUND Success of allogeneic hematopoietic cell transplantation (alloHCT) is sharped by alloreactivity, i.e. the ability of donor T-cells to recognize particular recipient motifs as non-self and get activated. Donor T-cells recognizing recipient tissue can cause harmful graft-versus-host disease (GvHD) while activity against leukemic blasts can...

Desmoplastic small round cell tumor (DSRCT) is a rare, radiosensitive, yet difficult-to-treat sarcoma subtype affecting predominantly male adolescents. Extensive intraperitoneal seeding is common and requires multimodal management. With no standard therapy established, the prognosis remains poor, and new treatment options are needed. We demonstrate...

Background: Due to the high risk of severe infection among pediatric hematology and oncology patients, antimicrobial use is particularly high. With our study, we quantitatively and qualitatively evaluated, based on institutional standards and national guidelines, antimicrobial usage by employing a point-prevalence survey with a multi-step, expert...

Histopathological differentiation in pediatric adrenocortical carcinoma (pACC) is difficult and clinical prediction and stratification scores are not evaluated yet. Therefore, this review aims to summarize current evidence on the value and accuracy of the two commonly used scoring systems (Weiss/AFIP) pACC. On this base, one might be able to evalua...

Objectives Initial treatment in patients with haemophilia remains challenging. The choice of therapy, timing, dose and frequency have been and are still under intense debate. New treatment options like novel factor concentrates and non-factor therapies broaden the discussion. Design The German Paediatric Haemophilia Research Database (GEPHARD) is a...

Objective Adrenal resections are rare procedures of a heterogeneous nature. While recent European guidelines advocate a minimum annual caseload for adrenalectomies (6 per surgeon), evidence for a volume–outcome relationship for this surgery remains limited. Design A retrospective analysis of all adrenal resections in Germany between 2009 and 2017...

Objective Pediatric adrenocortical carcinoma (pACC) is rare and prognostic stratification remains challenging. We summarized clinical prognostic factors of pACC and determined the prognostic value of the pediatric scoring system (pS-GRAS) in adaption to the recommendation (S-GRAS) of ENSAT for the classification of adult ACC. Design Analysis on pAC...

Background Adrenalectomies are rare procedures especially in childhood. So far, no large cohort study on this topic has been published with data on to age distribution, operative procedures, hospital volume and operative outcome.Methods This is a retrospective analysis of anonymized nationwide hospital billing data (DRG data, 2009-2017). All adrena...

Purpose In selected cases of severe Cushing’s syndrome due to uncontrolled ACTH secretion, bilateral adrenalectomy appears unavoidable. Compared with unilateral adrenalectomy (for adrenal Cushing’s syndrome), bilateral adrenalectomy has a perceived higher perioperative morbidity. The aim of the current study was to compare both interventions in end...

Introduction EBV associated lymphoproliferative disorders (EBV LPD) are a known complication following solid organ or hematopoietic stem cell transplantation. The disturbance of the immune system leads to a lack of control of latent EBV-infected B-cells, as control by T-cells is mandatory to prevent uninhibited cell proliferation. EBV LPD in other...

Background and purpose Pediatric adrenocortical carcinoma (pACC) is a rare disease with poor prognosis. Publications on radiotherapy (RT) are scarce. This review summarizes the current data on RT for pACC and possibly provides first evidence to justify its use in this setting. Materials and Methods We searched the PubMed and Embase database for ma...

Background Allogeneic hematopoietic stem cell transplantation (allo-HSCT) has improved survival in high-risk childhood leukemia but is associated with long-term sequelae such as impaired pulmonary function and reduced exercise capacity impacting quality of life. Methods A convenience sample of 17 patients after allo-HSCT (HSCT—12 male, age 15.7±6....

ALK-positive histiocytosis is a rare subtype of histiocytic neoplasm first described in 2008 in three infants with multisystemic disease involving the liver and hematopoietic system. This entity has subsequently been documented in case reports and series to occupy a wider clinicopathologic spectrum with recurrent KIF5B-ALK fusions. The full clinico...

Adrenocortical tumors are rare in children. This systematic review summarizes the published evidence on pediatric adrenocortical carcinoma (ACC) to provide a basis for a better understanding of the disease, investigate new molecular biomarkers and therapeutic targets, and define which patients may benefit from a more aggressive therapeutic approach...

Rosai-Dorman-Disease is one subgroup of Non-Langerhans cell histiocytosis with massive lymphadenopathy. It is a generally a benign and self-limiting disease, but an association to hemato-lymphoid malignancies or immune diseases is suspected. We present the case of a 6-year old male patient with multifocal extranodal RDD developing a secondary biphe...

Background Immune thrombocytopenia (ITP) is an autoimmune disease associated with isolated thrombocytopenia, which is caused by an imbalance between platelet production and platelet destruction. Petechial and mucous membrane hemorrhages are characteristic of ITP, but life-threatening bleeding rarely occurs. Depending on the bleeding symptoms, ITP c...

Background Lipoblastoma is a rare benign mesenchymal neoplasm of infancy that most commonly occurs on the extremities and trunk but can arise at variable sites of the body. Retroperitoneal lipoblastomas are particularly rare but can grow to enormous size, and preoperative diagnosis is difficult with diverse, mostly malignant differential diagnoses...

Patient: Male, 3-day-old Final Diagnosis: Neonatal leukemia Symptoms: Skin lesions Medication: — Clinical Procedure: Biopsy • stem cell transplant Specialty: Hematology • Pediatrics and Neonatology Objective Rare disease Background Neonatal acute leukemia is a rare condition. Little is known about its incidence and outcomes, and treatment options...

Viral reactivation occurs frequently in the context of immunodeficiency and immunosuppression after allogeneic hematopoietic stem cell transplantation (allo-HSCT) and can cause severe complications. The aim of this single-center retrospective analysis was to characterize viral infections in the first year after HSCT, to investigate risk factors and...

Defects of platelet intracellular signaling can result in severe platelet dysfunction. Several mutations in each of the linked genes FERMT3 and RASGRP2 on chromosome 11 causing a Glanzmann‐like bleeding phenotype have been identified so far. We report on novel variants in two unrelated pediatric patients with severe bleeding diathesis—one with leuk...

Interleukin 10 is a central regulator of the antigen-presenting function of myeloid cells. It exerts immunomodulatory effects in vivo and induces a regulatory phenotype in monocyte-derived cells in vitro. We analyzed phenotype and function of monocytic cells in vitro in relation to the cytokine milieu and the timing of TLR-based activation. In GM-C...

Hematopoietic stem cell transplantation (HSCT) is a life-saving procedure for children with a variety of malignant and nonmalignant conditions. However, even if immune reconstitution after HSCT has been studied extensively, until now, data on the comparison of immune reconstitution after autologous versus allogeneic HSCT are scarce, but might provi...

Background: Hematopoietic stem cell transplantation (HSCT) is a life-saving procedure for children with a variety of (non) malignant conditions. GvHD is a severe complication with high morbidity and mortality. The pathogenesis remains unclear. We studied dendritic cell (DC) reconstitution to detect potential differences, which may improve our know...

Hematopoietic stem cell transplantation (HSCT) has been an effective method for treating a wide range of malignant or non-malignant disorders. In case of an autologous HSCT, patients receive their own stem cells after myeloablation before extraction. Allogeneic HSCT uses stem cells derived from a donor. Despite being associated with a high risk of...

Declaration of selected complications following allogeneic or autologous HSCT. (DOCX)

Inherited platelet disorders (IPD) form a rare and heterogeneous disease entity that is present in about 8% of patients with non-acquired bleeding diathesis. Identification of the defective cellular pathway is an important criterion for stratifying the patient's individual risk profile and for choosing personalized therapeutic options. While costs...

B cells and DCs are suspected to play an important role in the pathogenesis of cGvHD, which is a serious complication of HSCT with high morbidity. It is characterized by immune responses of donor immune cells against recipient-derived antigens. athogenesis is not yet fully understood, however reconstitution of B cells after HSCT has similarities to...

Invasive fungal infections with Aspergillus species, mostly Aspergillus fumigatus, are a frequently occurring event after allogeneic stem cell transplantation (alloSCT) also in the paediatric setting. As in adults, invasive aspergillosis (IA) in children is associated with a poor prognosis and requires intensive and prolonged treatment. Diagnostic...

No abstract is available for this article.

No abstract is available for this article.

Combined biomarker screening is increasingly used to diagnose invasive aspergillosis (IA) in high-risk patients. In adults, the combination of galactomannan (GM) and fungal DNA detection has proven to be beneficial in the diagnosis of IA. Data in purely paediatric cohorts is scarce. Here, we monitored 39 children shortly before and after allogeneic...

Background: Hermansky-Pudlak syndrome (HPS) is a rare autosomal recessive disease characterized by oculocutaneous albinism and platelet dysfunction. We report on a novel HPS6 homozygous frameshift variant (c.1919_1920delTC; p.Val640Glyfs*29) in a nonconsanguineous Caucasian family with two affected siblings (index patients) who presented with ocul...

Varicella in oncology patients can result in serious complications. We analyzed trends in hospitalization rates and characteristics of pediatric oncology and non-oncology patients hospitalized with varicella during the first 7 years after introduction of routine varicella vaccination. Our data included children <17 years of age with an Internationa...

Background: Glanzmann thrombasthenia (GT) is an autosomal recessive bleeding disorder. Platelets from patients with GT show quantitative and/or qualitative defects of the platelet membrane glycoprotein (GP) IIb/IIIa complex, also called integrin αIIbβ3. On activated platelets the αIIbβ3 binds von fibrinogen and Willebrand factor which leads to plat...

Introduction: Considering that cytokines play an important role in immune response and that many infectious, autoimmune and malignant diseases are influenced by cytokine production, we hypothesized that genetically determined cytokine gene polymorphism might have an important influence on prognosis in pediatrics acute lymphoblastic leukemia (ALL)....

Introduction: Transplantation of haematopoietic stem cell (HSCT) from human leucocyte antigen (HLA)-disparate parental donors presents an approach for the treatment of patients lacking an HLA-matched donor, but little is known about differences in immune reconstitution as compared to conventional BMT. We prospectively compared paediatric recipients...

Introduction: Inadequate lymphocyte functions are an important clinical problem complicating chemotherapy by severe bacterial and fungal infections. During an ongoing immune response, cytokines produced by T-cells are critical to the effectiveness of that response. To understand what happens with the immune system in the beginning of therapy, we in...