In the light of the dynamics of fertility impairment and recovery in the HSCT patients reviewed, these patients should be counselled comprehensively regarding fertility preservation measures.

Significant advances in supportive care for patients with transfusion-dependent thalassemia major (TDT) have improved patients' life expectancy. However, transfusion-associated iron overload remains a significant barrier to long-term survival with good quality of life. Today, allogeneic hematopoietic stem cell transplantation (HSCT) is the current curative standard of care. Alongside selection of the best available donor, an optimized conditioning regimen is crucial to maximize outcomes for patients with TDT undergoing HSCT. The aim of this retrospective analysis was to investigate the role of busulfanfludarabine-based and treosulfan-fludarabine-based conditioning in TDT patients undergoing HSCT. We included 772 patients registered in the European Society for Blood and Marrow Transplantation (EBMT) database who underwent first HSCT between 2010 and 2018. Four hundred ten patients received busulfan-fludarabine-based conditioning (median age 8.6 years) and 362 patients received treosulfan-fludarabine-based conditioning (median age 5.7 years). Patient outcomes were retrospectively compared by conditioning regimen. Two-year overall survival was 92.7% (95% confidence interval: 89.3-95.1%) after busulfan-fludarabine-based conditioning and 94.7% (95% confidence interval: 91.7-96.6%) after treosulfan-fludarabine-based conditioning. There was a very low incidence of second HSCT overall. The main causes of death were infections, graft-versus-host disease, and rejection. In conclusion, use of busulfan or treosulfan as the backbone of myeloablative conditioning for patients with TDT undergoing HSCT resulted in comparably high cure rates. Long-term follow-up studies are warranted to address the important issues of organ toxicities and gonadal function.

Background A growing number of cancer and hematopoietic stem cell transplant (HSCT) survivors require long-term follow-up with optimal communication schemes, and patients' compliance is crucial. Adolescents have various unmet needs. Regarding self-report of symptoms and health status, users of mobile apps showed enhanced compliance. Currently, HSCT aftercare at the HSCT outpatient clinic of the St. Anna Children’s Hospital in Vienna, Austria, is based on handwritten diaries, carrying various disadvantages. Recently, we developed the prototype of a web-based, self-monitoring gamified mobile app tailored for adolescents: the INTERACCT (Integrating Entertainment and Reaction Assessment into Child Cancer Therapy) app. Objective This observational, prospective study evaluated the usability of the INTERACCT app for tracking real-time self-reported symptoms and health status data in adolescent HSCT patients and a healthy matched control group. The primary outcome of the study was the quality of the self-reported medical information. We hypothesized that the mobile app would provide superior medical information for the clinicians than would the handwritten diaries. Methods Health data were reported via paper diary and mobile app for 5 consecutive days each. The quality of medical information was rated on a 5-point scale independently and blinded by two HSCT clinicians, and the duration of use was evaluated. A total of 52 participant questionnaires were assessed for gaming patterns and device preferences, self-efficacy, users’ satisfaction, acceptability, and suggestions for improvement of the mobile app. Interrater reliability was calculated with the intraclass correlation coefficient, based on a two-way mixed model; one-way repeated-measures analysis of variance and t tests were conducted post hoc. Descriptive methods were used for correlation with participants’ demographics. For users’ satisfaction and acceptability of the mobile app, the median and the IQR were calculated. Results Data from 42 participants—15 patients and 27 healthy students—with comparable demographics were evaluated. The results of our study indicated a superiority of the quality of self-reported medical data in the INTERACCT app over traditional paper-and-pencil assessment (mobile app: 4.14 points, vs paper-based diary: 3.77 points, P=.02). The mobile app outperformed paper-and-pencil assessments mainly among the patients, in particular among patients with treatment-associated complications (mobile app: 4.43 points, vs paper-based diary: 3.73 points, P=.01). The mobile app was used significantly longer by adolescents (≥14 years: 4.57 days, vs ≤13 years: 3.14 days, P=.03) and females (4.76 days for females vs 2.95 days for males, P=.004). This corresponds with a longer duration of use among impaired patients with comorbidities. User satisfaction and acceptability ratings for the mobile app were high across all groups, but adherence to entering a large amount of data decreased over time. Based on our results, we developed a case vignette of the target group. Conclusions Our study was the first to show that the quality of patient-reported medical information submitted via the INTERACCT app embedded in a serious game is superior to that submitted via a handwritten diary. In light of these results, a refinement of the mobile app supported by a machine learning approach is planned within an international research project.

Thyroid disorders are well-studied after allogeneic haematopoietic stem cell transplantation (HSCT) following total body irradiation (TBI)-based conditioning, occurring in 15–30% of paediatric survivors. The toxic effect of TBI is known but data on the role of immunological dysregulation (ID) and chronic graft-versus-host-disease (cGvHD) are scarce. We studied functional and structural thyroid disorders in 97 paediatric ALL patients after TBI-based HSCT, assessing their correlation with patient/transplant characteristics including cGvHD, prolonged immunosuppression and ID. The 10- and 15-year cumulative incidence (CI) of functional disorders was 50 and 60%. Univariate analysis revealed TBI in 6 vs. 8 fractions (p = 0.01), an interval between ALL diagnosis and HSCT <1 year (p = 0.038), and the application of ATG (p = 0.044) as risk factors. The 10- and 15-year CI of structural disorders was 60 and 80%. No correlation between patient/transplant characteristics and structural disorders was observed. cGvHD, prolonged immunosuppression and additional radiotherapy were not associated with any thyroid disease. We observed a significant correlation between ID and the development of thyroid dysfunction in patients with structural changes (10-year CI: 77% for patients with ID vs. 56% without ID, p = 0.02). The impact of our results on thyroid follow-up evaluations and the significance of hormonal replacement therapy are discussed.