Long-Term Immune Recovery After Hematopoietic Stem Cell Transplantation for ADA Deficiency: a Single-Center Experience.

Unconditioned hematopoietic stem cell transplantation (HSCT) is the recommended treatment for patients with adenosine deaminase (ADA)-deficient severe combined immunodeficiency with an HLA-matched sibling donor (MSD) or family donor (MFD). Improved overall survival (OS) has been reported compared to the use of unrelated donors, and previous studies have demonstrated that adequate cellular and humoral immune recovery can be achieved even in the absence of conditioning. Detailed insight of the long-term outcome is still limited. We aim to address this by studying a large single-center cohort of 28 adenosine deaminase-deficient patients who underwent a total of 31 HSCT procedures, of which more than half were unconditioned. We report an OS of 85.7% and event-free survival of 71% for the entire cohort, with no statistically significant differences after procedures using related or unrelated HLA-matched donors. We find that donor engraftment in the myeloid compartment is significantly diminished in unconditioned procedures, which typically use a MSD or MFD. This is associated with poor metabolic correction and more frequent failure to discontinue immunoglobulin replacement therapy. Approximately one in four patients receiving an unconditioned procedure required a second procedure, whereas the use of reduced intensity conditioning (RIC) prior to allogeneic transplantation improves the long-term outcome by achieving better myeloid engraftment, humoral immune recovery, and metabolic correction. Further longitudinal studies are needed to optimize future management and guidelines, but our findings support a potential role for the routine use of RIC in most ADA-deficient patients receiving an HLA-identical hematopoietic stem cell transplant, even when a MSD or MFD is available.

Use of digital media for the education of health professionals in the treatment of childhood asthma / Uso de mídia digital na educação de profissionais de saúde para tratamento da asma infantil

OBJECTIVES: Inhalation therapy is the main treatment for asthma and its adequate use has been a factor responsible for disease control; therefore, the aim of the study was to determine whether a digital media tool, which features portability on mobile phones, modifies the assimilation of the inhalation technique. METHODS: A total of 66 professionals working in the health care area with the pediatric population were selected. They were submitted to a pre-test on their knowledge of inhalation therapy. The professionals were randomized into two groups (A and B). Group A received a media application on their mobile phones showing the steps of inhalation therapy, while group B received the same information in written form only. A post-test was applied after 15 days. The results (pre- and post-) were analyzed by two pediatric pulmonologists. RESULTS: Of the 66 professionals, 87.9% were females. Of a total possible score of ten, the mean score obtained in the pre-test was 5.3 ± 3, and in the second test, 7.5 ± 2 (p < 0.000). There were no significant differences when comparing the two groups (p = 0.726). The nurses had the lowest mean scores in the initial test (2.3 ± 2); however, they were the group that learned the most with the intervention, showing similar means to those of other groups in the second test (6.1 ± 3). CONCLUSION: There was significant improvement in knowledge about inhalation therapy in all professional categories using both methods, demonstrating that education, when available to professionals, positively modifies medical practice. .

OBJETIVOS: A inaloterapia representa a principal forma de tratamento da asma e seu uso adequado tem sido fator responsável pelo controle da doença. Desse modo, o objetivo do estudo foi determinar se uma ferramenta de mídia digital, dotada de portabilidade na forma de telefonia móvel, modifica a assimilação da técnica inalatória. MÉTODOS: Foram selecionados 66 profissionais que atuam na área da saúde com população pediátrica e submetidos a um pré-teste sobre seus conhecimentos de inaloterapia. Os profissionais foram randomizados em dois grupos (A e B). O grupo A recebeu em seu telefone móvel um aplicativo de mídia com os passos da inaloterapia, enquanto o grupo B recebeu as mesmas informações apenas de forma escrita. Após 15 dias, fez-se um pós-teste. Os resultados (pré e pós) foram analisados por dois pneumologistas pediátricos. RESULTADOS: Dos 66 profissionais, 87,9% eram do sexo feminino. Num escore total possível de 10, a média das notas obtidas no pré-teste foi de 5,3 ± 3 e as do segundo teste 7,5 ± 2 (p < 0,000). Não houve diferenças significativas na comparação os dois grupos (p = 0,726). Os profissionais de enfermagem apresentaram a menor média nas provas iniciais (2,3 ± 2), porém foi o grupo que aprendeu mais com a intervenção e apresentou média similar aos outros grupos na segunda prova (6,1 ± 3). CONCLUSÃO: Houve melhoria significativa no conhecimento sobre inaloterapia em todas as categorias profissionais com o uso de ambos os métodos. Isso comprovou que a educação, quando oferecida aos profissionais, modifica positivamente a prática médica. .

Use of digital media for the education of health professionals in the treatment of childhood asthma.

OBJECTIVES: Inhalation therapy is the main treatment for asthma and its adequate use has been a factor responsible for disease control; therefore, the aim of the study was to determine whether a digital media tool, which features portability on mobile phones, modifies the assimilation of the inhalation technique. METHODS: A total of 66 professionals working in the health care area with the pediatric population were selected. They were submitted to a pre-test on their knowledge of inhalation therapy. The professionals were randomized into two groups (A and B). Group A received a media application on their mobile phones showing the steps of inhalation therapy, while group B received the same information in written form only. A post-test was applied after 15 days. The results (pre- and post-) were analyzed by two pediatric pulmonologists. RESULTS: Of the 66 professionals, 87.9% were females. Of a total possible score of ten, the mean score obtained in the pre-test was 5.3 ± 3, and in the second test, 7.5 ± 2 (p<0.000). There were no significant differences when comparing the two groups (p=0.726). The nurses had the lowest mean scores in the initial test (2.3 ± 2); however, they were the group that learned the most with the intervention, showing similar means to those of other groups in the second test (6.1 ± 3). CONCLUSION: There was significant improvement in knowledge about inhalation therapy in all professional categories using both methods, demonstrating that education, when available to professionals, positively modifies medical practice.