Central nervous system (CNS) tumors are the second most common pediatric malignancies after acute leukemias and are the most common pediatric solid tumors. Although cure rates have improved with ...numerous technical advances in multimodal therapy, the prognosis remains poor for some high-risk histological type and for patients with residual, recurrent or disseminated disease. Radiotherapy (RT) remains an integral part of treatment for childhood brain tumors; however, the profound and irreversible sequelae of brain irradiation in the younger children are now well documented. In an effort to decrease irradiation toxicity while improving survival and quality of life in these patients, high-dose chemotherapy with autologous hematopoietic stem cell transplantation (HD-CT&autoHSCT) has been incorporated in both up-front as well as recurrent therapies. In up-front treatment, it is used in patients under the age of 3 years to delay RT or not to use RT at all. It can be used tandem non-myeloablatively in patients older than 3 years of age, after dose-intensive chemotherapy, both to shorten the neutropenic period and to give more intense chemotherapy in a shorter time when compared to conventional chemotherapy treatment approaches. AutoHSCT may also be considered after a myeloablative conditioning regimen for relapsed embryonal brain tumors, as either once or tandem, in cases with good response to salvage therapy as consolidation. In this talk, the role of autoHSCT in childhood brain tumors will be discussed by giving the results from international studies.
Background
Despite the increasing performance of allogeneic hematopoietic cell transplantation over the last decades, graft‐versus‐host disease (GVHD) remains the main cause of morbidity and ...mortality. The efficacy of ruxolitinib against GVHD has been demonstrated in adult studies; however, very few studies have been conducted in children.
Procedure
This study aimed to evaluate the efficacy of ruxolitinib in 29 children with steroid‐refractory acute or chronic GVHD. Twenty‐five (87%) patients received at least three different immune modulator agents, including methylprednisolone, before initiating ruxolitinib.
Results
All grade 2 acute GVHD patients completely responded to ruxolitinib treatment; 82% of high‐grade (3‐4) acute GVHD patients and 80% of chronic GVHD (moderate‐severe) patients had at least a partial response. Of seven patients with bronchiolitis obliterans, five had a partial response after ruxolitinib. Of 29 patients, 22 were administered steroids at any time in the first month of acute GVHD or the first three months of chronic GVHD during ruxolitinib usage, which was significantly tapered by the end of the observation period.
Conclusion
Steroid‐refractory acute and chronic pediatric GVHD patients treated with ruxolitinib had a high overall response rate, with the additional benefit of steroid sparing.
Background
ALD is a rare X‐linked peroxisomal metabolic disorder with many distinct phenotypes of disease that emerge on a wide scale from adrenal insufficiency to fatal cALD which progresses to a ...vegetative state within a few years. Currently, HSCT is the only treatment method known to stabilize disease progression in patients with cALD. In this study, we aim to report our HSCT experience in patients with cALD and the factors that determine the success of HSCT, as a single‐center experience.
Methods
The study cohort involves 23 boys with cALD and three patients with ALD trait and new‐onset abnormal behavior who underwent allogeneic HSCT between January 2012 and September 2019 in our transplantation center. Loes scoring, NFS, scale and MFD were performed for evaluating the severity of the cerebral disease. The study cohort was divided into two groups according to baseline NFS and Loes score: early‐stage (NFS ≤ 1 and Loes score <9) and advanced stage (NFS > 1 or Loes score ≥9).
Results
The pretransplant stage of disease impacted both OS and MFD‐free survival. The estimated OS and MFD‐free survival at 3 years in patients with advanced disease were 46.1% (95% CI 19.0–73.2) and 23.1% (95% CI 0.2–46.0), respectively, and all patients with the early disease were alive (p: .004) and MFD‐free (p < .001) at 3 years.
Conclusion
This study demonstrated that early HSCT is vital in patients with cALD. The early‐stage disease had a significant survival advantage and free from disease progression after HSCT.
Background
Post‐Cy administration for GVHD prophylaxis in unmanipulated haploidentical HSCT has resulted in improved outcomes in recent years. Studies in children are lacking and accordingly we ...present the outcomes of 62 haploidentical transplantation for high‐risk children.
Procedure
We retrospectively assessed 62 transplants in 60 patients who underwent haploidentical‐related HSCT with unmanipulated stem cells and for whom Post‐Cy was used for GVHD prophylaxis.
Results
Myeloid reconstitution was achieved on day + 30 for 57 of the 62 patients. The median follow‐up of the surviving 39 patients (63%) was 26 months, with a range of 6‐57 months. The OS and EFS at 2 years were 64.6% (52.0%‐77.2%, 95% CI) and 58.9% (46.1%‐71.7%, 95% CI), respectively. The only factor in our multivariate analysis that contributed to an inferior EFS was a poor remission status prior to HSCT (HR, 8.30; 1.08‐63.56; P = 0.041, 95% CI).
Conclusion
The results of T‐cell replete haploidentical transplantation with Post‐Cy GVHD prophylaxis in high‐risk pediatric patients are promising. However, further research is needed to determine the factors that have affect HLA compatibility for predicting the success of haploidentical transplantations.
The aims were to investigate the incidence of BKV infection and the presence of HC in pediatric patients undergoing HSCT. Twenty‐four children patients (M/F: 17/7) undergoing HSCT in a single center ...over a period of 1 year were included in the study. The presence of BKV DNA was determined by quantitative real‐time PCR in plasma and urine samples at the following times: before transplantation, twice a week until engraftment time, and weekly for + 100 days. The mean age of the patients was 7.79 ± 5.03 years, the mean follow‐up time was 95.6 ± 25.9 days, and the average number of samples per patient was 15.8 ± 3.2. BKV DNA was detected in at least one urine sample in 91.6% (n: 22) and at least one plasma sample in 75% (n:18) of the patients. The median time to the first BKV DNA positivity in urine and plasma samples was 11 (range: 1‐80) and 32 days (range: 2‐79), respectively. The median value of BKV DNA copies in urine and plasma were 1.7 × 106 (range: 2.8 × 101‐1.2 × 1014) and 1.9 × 103 copies/mL (range: 3‐2.1 × 106), respectively. Thirteen patients (54.2%) had hematuria with BKV viruria; 8 (33.3%) patients had viremia. The median value of the BKV DNA copies in urine and plasma was 4.4 × 107 (range: 65‐1 × 1011) and 2.9 × 103 (range: 7‐7.8 × 104) copies/mL in these patients. Two (15.4%) of the 13 patients with BKV viruria and hematuria were diagnosed with BKV‐related HC. BKV DNA viral load monitoring of urine and plasma in pediatric HSCT patients with a high risk for viral infections is valuable for understanding the development of BKV–related HC.
Hematopoietic stem cell transplantation (HSCT) is a curative therapy option for hematologic malignancies. Iron overload is common in this patient group and can impact short-term and long-term ...nonrelapse mortality.
Retrospective observational cohort study.
To evaluate the effect of iron load on early and late HSCT outcomes in patients with acute leukemia and myelodysplasia to assess the necessity of reducing iron load.
Sixty patients who underwent HSCT in pediatric stem cell transplantation unit between 2000 and 2012 were evaluated retrospectively. The patients were divided into those with pretransplantation serum ferritin levels above and below the median value of 1299 ng/mL.
Forty-two (70%) of the patients were male, mean ages of the low and high ferritin groups were 85.43±9.42 and 118.56±10.04 months, respectively. Acute graft-versus-host disease (GVHD) within the first 100 days and acute liver GVHD were significantly more common in the high ferritin group (P<0.011 for both). Ferritin level was not associated with rates of engraftment syndrome, veno-occlusive disease, early/late infection, relapse, or overall and disease-free survival.
In our study, significant result especially in terms of acute liver GVHD, was important to emphasize the need to be more careful in terms of acute liver GVHD risk in early liver pathologies in patients with high levels of ferritin after transplantation. In future large studies may be helpful to explain the relationship between acute liver GVHD and high ferritin levels.
Despite poor survival, controversies remain in the treatment for refractory or relapsed pediatric non-Hodgkin lymphoma (r/r NHL). The current project aimed to collect international experience on the ...re-induction treatment of r/r NHL, hematopoietic stem cell transplantation (HSCT), risk factors associated with outcome, and to suggest treatment recommendations. Inclusion criteria were (i) refractory disease, disease progression or relapse of any NHL subtype except anaplastic large cell lymphoma, (ii) age < 18 years at initial diagnosis, (iii) diagnosis in/after January 2000. Data from 639 eligible patients were evaluable. The eight-year probability of overall survival was 34 ± 2% with highly significant differences according to NHL subtypes: 28 ± 3% for 254 Burkitt lymphoma/leukemia, 50 ± 6% for 98 diffuse large B-cell lymphomas, 57 ± 8% for 41 primary mediastinal large B-cell lymphomas, 27 ± 3% for 177 T-lymphoblastic lymphomas, 52 ± 10% for 34 precursor-B-cell lymphoblastic lymphomas and 30 ± 9% for 35 patients with rare NHL subtypes. Subtype-specific factors associated with survival and treatment recommendations are suggested. There were no survivors without HSCT, except in few very small subgroups. Conclusions: There is an urgent need to further improve survival in r/r NHL. The current study provides the largest real-world series, which underlines the role of HSCT and suggests treatment recommendations.
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