ABSTRACT
Factor VIII (FVIII) inhibitors remain a serious complication of treatment for patients with haemophilia A. Immune tolerance induction (ITI) can eliminate inhibitors in the majority of patients, but there are major concerns related with this therapy. Investigators have raised the possibility that the use of FVIII/von Willebrand factor (FVIII/VWF) concentrates may improve the success rate of ITI and may shorten the duration of therapy necessary to attain tolerance. This retrospective study describes 25 patients at five institutions in the USA, who were treated with FVIII/VWF concentrate as part of their ITI. These were all patients who were considered poor prognosis because of clinical and laboratory characteristics, which made ITI less likely to be successful or because of a poor response to initial ITI with a monoclonal/recombinant FVIII concentrate. Overall success (complete tolerization) was 32% with another 40% attaining partial tolerization, but not complete tolerization. Of those patients attaining only partial tolerization, two patients ultimately discontinued ITI and had return of their high titre inhibitors. Eight percent of patients failed to attain either partial or complete tolerization and discontinued ITI. Another 24% are continuing with ITI but have titres of >10 BU. This study adds further retrospective data to the information regarding the use of FVIII/VWF concentrate in ITI.
Subject(s)
Factor VIII/therapeutic use , Hemophilia A/drug therapy , Immune Tolerance/drug effects , von Willebrand Factor/therapeutic use , Antibodies/blood , Child , Child, Preschool , Hemophilia A/immunology , Humans , Infant , Retrospective Studies , Treatment OutcomeSubject(s)
Catheterization, Peripheral , Hemophilia A/therapy , Infusion Pumps, Implantable , Adolescent , Catheterization, Peripheral/adverse effects , Catheterization, Peripheral/methods , Child , HIV Infections/complications , HIV-1 , Hemophilia A/complications , Humans , Male , Retrospective Studies , Treatment OutcomeABSTRACT
Pediatric cancer patients often become anxious, agitated, combative, and uncooperative due to the pain or fear of pain during invasive procedures. Generally, it is not the actual administration of medicines that produces this reaction, but the fear of the needle stick itself. Increased education and implementation of coping mechanisms is often not enough to allay this fear. The tangible solution of using ethyl chloride, an anesthetic spray, before port sticks, lumbar punctures, and bone marrow aspirations, was instituted by the hematology-oncology clinic to determine if the pain, emotional trauma, and fear of cancer treatments could be reduced in oncology patients. Survey results on 60 patients and 60 parents/caretakers showed that when given the choice to use the spray or to refuse its use, 68% of the parents thought that the patient had more of a sense of control and, thus, involvement in their treatment. Seventy-eight percent of the patients reported experiencing less pain associated with procedures. Staff noted an increase in cooperation, less combativeness, and more compliance with treatment. Perceiving the child's discomfort diminished, 87% of the parents/caretakers report feeling less anxious and, therefore, more capable of being supportive to each other and their child. These results verified the staff's perceptions of the advantages of using this noninvasive anesthetic. Ethyl chloride is an easy, effective, concrete approach to reducing procedural pain in pediatric oncology patients.
