Case Report: Lessons Learned From Subsequent Autologous and Allogeneic Hematopoietic Stem Cell Transplantations in a Pediatric Patient With Relapsing Polychondritis

Background: Autologous hematopoietic stem cell transplantation (autoHSCT) is increasingly being recognized as a treatment option for severe refractory autoimmune diseases (AD). However, efficacy is hampered by high relapse rates. In contrast, allogeneic HSCT (alloHSCT) has high potential to cure AD, but is associated with significant morbidity and mortality, and data in AD are limited. Experience with autoHSCT in relapsing polychondritis, a rare episodic inflammatory disorder characterized by destruction of cartilage, is scarce and alloHSCT has not been described before. Case Presentation: Here, we present a case of a 9-year-old girl who was diagnosed with relapsing polychondritis, with severe airway involvement requiring a tracheostomy. The disease proved to be steroid-dependent and refractory to a wide array of disease-modifying anti-rheumatic drugs and biologicals. After an autoHSCT procedure, the disease became inactive for a short period of time, until the patient experienced a relapse after 31 days, accompanied by repopulation of effector/memory CD8+ T cells. Because of persistent inflammation and serious steroid toxicity, including severe osteoporosis, growth restriction, and excessive weight gain, the patient was offered an alloHSCT. She experienced transient antibody-mediated immune events post-alloHSCT, which subsided after rituximab. She ultimately developed a balanced immune reconstitution and is currently still in long-term disease remission, 8 years after alloHSCT. Conclusion: This case adds to the few existing reports on autoHSCT in relapsing polychondritis and gives new insights in its pathogenesis, with a possible role for CD8+ T cells. Moreover, it is the first report of successful alloHSCT as a treatment for children with this severe autoimmune disease

Correction to : A phase I/II study of gemcitabine during radiotherapy in children with newly diagnosed diffuse intrinsic pontine glioma (Journal of Neuro-Oncology, (2017), 135, 2, (307-315), 10.1007/s11060-017-2575-9)

In Table 2 of the original publication, there were errors in the baseline scores for the PedsQL TM 3.0 Cancer Module questionnaire, so a corrected version of Table 2 is shown in this erratum. In the subcategories of the PedsQL TM 3.0 Cancer Module questionnaire, nausea and fear of procedure did not score significantly lower after treatment compared to baseline. So, based on the corrected data in Table 2, there was no significant decrease in the total score of the cancer questionnaire, and this statement in the previous manuscript was incorrect

Correction to : A phase I/II study of gemcitabine during radiotherapy in children with newly diagnosed diffuse intrinsic pontine glioma (Journal of Neuro-Oncology, (2017), 135, 2, (307-315), 10.1007/s11060-017-2575-9)

In Table 2 of the original publication, there were errors in the baseline scores for the PedsQL TM 3.0 Cancer Module questionnaire, so a corrected version of Table 2 is shown in this erratum. In the subcategories of the PedsQL TM 3.0 Cancer Module questionnaire, nausea and fear of procedure did not score significantly lower after treatment compared to baseline. So, based on the corrected data in Table 2, there was no significant decrease in the total score of the cancer questionnaire, and this statement in the previous manuscript was incorrect

External validation of the diffuse intrinsic pontine glioma survival prediction model: a collaborative report from the International DIPG Registry and the SIOPE DIPG Registry

We aimed to perform external validation of the recently developed survival prediction model for diffuse intrinsic pontine glioma (DIPG), and discuss its utility. The DIPG survival prediction model was developed in a cohort of patients from the Netherlands, United Kingdom and Germany, registered in the SIOPE DIPG Registry, and includes age <3 years, longer symptom duration and receipt of chemotherapy as favorable predictors, and presence of ring-enhancement on MRI as unfavorable predictor. Model performance was evaluated by analyzing the discrimination and calibration abilities. External validation was performed using an unselected cohort from the International DIPG Registry, including patients from United States, Canada, Australia and New Zealand. Basic comparison with the results of the original study was performed using descriptive statistics, and univariate- and multivariable regression analyses in the validation cohort. External validation was assessed following a variety of analyses described previously. Baseline patient characteristics and results from the regression analyses were largely comparable. Kaplan–Meier curves of the validation cohort reproduced separated groups of standard (n = 39), intermediate (n = 125), and high-risk (n = 78) patients. This discriminative ability was confirmed by similar values for the hazard ratios across these risk groups. The calibration curve in the validation cohort showed a symmetric underestimation of the predicted survival probabilities. In this external validation study, we demonstrate that the DIPG survival prediction model has acceptable cross-cohort calibration and is able to discriminate patients with short, average, and increased survival. We discuss how this clinico-radiological model may serve a useful role in current clinical practice

Additional file 2 of Meningococcal ACWY conjugate vaccine immunogenicity in adolescents with primary or secondary immune deficiencies, a prospective observational cohort study

Supplementary Material

Additional file 1 of Meningococcal ACWY conjugate vaccine immunogenicity in adolescents with primary or secondary immune deficiencies, a prospective observational cohort study

Supplementary Material