Transborder program of allogeneic hematopoietic cell transplantations from unrelated donors for Ukrainian children between 2015–2020 in Bydgoszcz

Introduction: The aim of this study was the analysis of the organizational aspects, treatments and outcomes of the program of allogeneic hematopoietic stem cell transplantations (HCT) from unrelated donors for children from Ukraine in the Transplant Center in Bydgoszcz, Poland over the period 2015–2020. Material and methods: Patients from Ukraine were referred via email by parents or by the Tabletochki Charity Foundation based in Kyiv directly to transplant physicians or to the Medical Office of Jurasz University Hospital 1 in Bydgoszcz. Results: Overall, 28 allo-matched unrelated donor-HCTs in 22 patients were performed. Children were diagnosed for malignant (n = 19) or non-malignant (n = 3) diseases. Most of the children were in advanced stages of the disease. The cumulative probabilities of hematological engraftment measured by neutrophil and platelet recovery were 90.6% and 77.3%, respectively. The cumulative incidence of acute graft-versus-host disease ≥2° and extensive chronic graft-versus-host disease were 40.9% and 27.7%, respectively. Overall, 11/19 patients with malignant diseases and 1/3 with non-malignant diseases were alive at the end of a median follow-up of 1.5 (range 0.1–6.5) years. Overall survival (OS) for all patients was 0.51 ± 0.11. Patients with malignancies at transplant standard risk had OS = 0.83 ± 0.15, while those with transplant high-risk had OS = 0.34 ± 0.15 (p = 0.025). A total of 10/22 children died. The treatment-related mortality rate was 6/22 (27.3%). The overall relapse rate among children with malignancies was 4/19 (21%). Conclusions: The program of transborder transplants for children from Ukraine was an important factor in international co-operation contributing to the establishment of a program of transplants from unrelated donors for children in Ukraine. The outcome was positive for more than half of the children referred to the program

Invasive fungal disease presenting as septic shock in immunocompromised pediatric and adult patients: summary of reported cases

Introduction: Septic shock is a very rare presentation of invasive fungal disease (IFD) in immunocompromised patients. The objective of this paper was to summarize reported cases of pediatric and adult patients with IFD presenting as septic shock in non-Candida infections. Literature data describing etiology, age, and outcome of septic shock as a presentation of IFD, is summarized. Material and methods: The available pediatric data included 23 patients, most of them with underlying non-hematological disease. Results: Only 6/23 (26.1%) were reported to survive this infection. Respective data in adults with invasive fungal disease presenting as septic shock were reported in 28 patients. Most of these patients were treated for acute leukemias (including three patients after hematopoietic cell transplantation); only 5/28 (17.9%) survived the infection. Conclusion: Invasive fungal disease presenting as septic shock in immunocompromised patients is a highly unusual presentation

Eltrombopag use in chronic immune thrombocytopenia of childhood: results from nationwide therapeutic program

BackgroundThrombopoietin receptor agonists have been repeatedly confirmed to be safe, efficient, and well tolerated in pediatric patients with chronic immune thrombocytopenia (cITP). Material and methodsIn this report, we present data summarizing the Polish experience of the use of eltrombopag in cITP patients, refractory to standard first-line care. Our analysis was based on clinical and epidemiological data from the Nationwide Therapeutic Program 2018–2020. Quality of the response to the eltrombopag treatment was defined according to the International Consensus Guidelines as follows: complete response (CR) defined as platelet count (PLT) ≥100 × 10/L and absence of bleeding; response (R) defined as PLT ≥30 × 10/L and at least two-fold increase in the baseline count and absence of bleeding. ResultsWe evaluated 60 patients (33 boys and 27 girls) with chronic and refractory ITP. Median age at beginning of treatment was 9.5 years. Median PLT at the first eltrombopag administration was 30 × 10/L. The median follow-up was 7 months (range, 3–22 months). After 1 week of treatment, response (R) was noted in 53.3% (95% confidence interval [CI]: 40.7%–66.0%) patients, and complete response (CR) was seen in 21.6% (95% CI: 11.2%–32.1%). We evaluated the long-term duration of the response and found that it was obtained in 84.4% (95% CI: 71.8%–97.0%) and 88.9% (95% CI: 77.0%–100%) of patients after 6 and 12 months, respectively, of eltrombopag therapy, while CR was reached, respectively, in 46.9% (95% CI: 29.6%–64.2%) and 29.6% (95% CI: 12.4%–46.9%) patients. No serious adverse events were reported. ConclusionOur data support the safety and efficacy of eltrombopag use in cITP pediatric patients

Efficacy of keratinocyte growth factor in prevention of oral mucositis in children undergoing allogeneic hematopoietic cell transplantation

IntroductionOral mucositis is regarded by patients as one of the worst and debilitating complications of conditioning and hematopoietic cell transplantation (HCT). Prevention of mucositis is one of the priorities of supportive therapy during and after conditioning. ObjectivesThe primary objective of the study was the analysis of efficacy of keratinocyte growth factor (KGF, palifermin) used in prophylaxis of oral mucositis in patients undergoing allo-HCT. The secondary objectives of the study included the analysis of the influence of palifermin on clinical course of oral mucositis and early transplant outcomes, as well as analysis of the contraindications of palifermin in patients undergoing allo-HCT. Patients and methodsA total number of 253 allo-HCT performed between 2003 and 2018 in patients aged 0–19 years in a single center were analyzed. Overall, in 161 HCTs, palifermin was administered. ResultsPatients receiving KGF were transplanted earlier in the context of calendar year, and more often received ATG, mainly due to the higher rate of unrelated donor transplants. Allo-HCT patients who were administered palifermin had shorter time of mucositis (median: 9 vs. 13 days, < 0.001), lower mucositis grade (median: 2° vs. 3°; < 0.001), shorter period of total parenteral nutrition (median: 19 vs. 22 days; = 0.018), and lower incidence of episodes of febrile neutropenia (median: 39.1% vs. 83.1%; < 0.001). ConclusionsThe use of palifermin has decreased duration and severity of oral mucositis in children after allo-HCT. Palifermin is a safe and well-tolerated compound in children undergoing allo-HCT

Very late relapse of ALL after 14 years: treatment with CAR-T cells

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