scholarly journals Hearing loss in patients with mucopolysaccharidoses ‐1 and ‐6 after hematopoietic cell transplantation: A longitudinal analysis

2020 ◽  
Vol 43 (6) ◽  
pp. 1279-1287 ◽  
Author(s):  
Brigitte T. A. Broek ◽  
Adriana L. Smit ◽  
Jaap Jan Boelens ◽  
Peter M. Hasselt
Keyword(s):  
Hearing Loss ◽  
Cell Transplantation ◽  
Longitudinal Analysis ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell

scholarly journals Longitudinal Analysis of Ocular Disease in Children with Mucopolysaccharidosis I after Hematopoietic Cell Transplantation

2020 ◽  
Vol 26 (5) ◽  
pp. 928-935 ◽  
Author(s):  
Brigitte T.A. van den Broek ◽  
Michelle B. van Egmond-Ebbeling ◽  
Jens A. Achterberg ◽  
Jaap Jan Boelens ◽  
Isa C. Vlessert ◽  
...  
Keyword(s):  
Cell Transplantation ◽  
Longitudinal Analysis ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Ocular Disease ◽  
Mucopolysaccharidosis I

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

2020 ◽  
Vol 51 (3) ◽  
pp. 172-178
Author(s):  
Natalia Bartoszewicz ◽  
Krzysztof Czyżewski ◽  
Robert Dębski ◽  
Anna Krenska ◽  
Ewa Demidowicz ◽  
...  
Keyword(s):  
Growth Factor ◽  
Cell Transplantation ◽  
Oral Mucositis ◽  
Hematopoietic Cell Transplantation ◽  
Clinical Course ◽  
Keratinocyte Growth Factor ◽  
Supportive Therapy ◽  
Primary Objective ◽  
Hematopoietic Cell ◽  
Unrelated Donor

AbstractIntroductionOral 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, p < 0.001), lower mucositis grade (median: 2° vs. 3°; p < 0.001), shorter period of total parenteral nutrition (median: 19 vs. 22 days; p = 0.018), and lower incidence of episodes of febrile neutropenia (median: 39.1% vs. 83.1%; p < 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.

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