BACKGROUND: Chronic granulomatous disease (CGD) is a primary immunodeficiency characterized by serious infections and inflammation. It can be managed conservatively with prophylactic antimicrobial agents or curatively with hematopoietic stem cell transplantation (HSCT). In the United Kingdom and Ireland there are cohorts of children managed both conservatively and curatively. OBJECTIVES: This study aimed to compare clinical outcomes (mortality and morbidity) in children managed conservatively and curatively. METHODS: Children were identified from specialist centers and advertising through special interest groups. Clinical data were collected from medical records regarding infections, inflammatory complications and growth, other admissions, and curative treatment. Comparisons were made for patients not undergoing HSCT and patients after HSCT. RESULTS: Seventy-three living children were identified, 59 (80%) of whom were recruited. Five deceased children were also identified. Clinical information was available for 62 children (4 deceased). Thirty (48%) children had undergone HSCT. Children who did not undergo transplantation had 0.71 episodes of infection/admission/surgery per CGD life year (95% CI, 0.69-0.75 events per year). Post-HSCT children had 0.15 episodes of infection/admission/surgery per transplant year (95% CI, 0.09-0.21 events per year). The mean z score for height and body mass index (BMI) for age was significantly better in post-HSCT children. Survival in the non-HSCT group was 90% at age 15 years. Survival in the post-HSCT group was 90%. CONCLUSIONS: Children with CGD not undergoing transplantation have more serious infections, episodes of surgery, and admissions compared with post-HSCT children. Children undergoing transplantation have better height for age. Survival is good at the end of the pediatric age range and also after HSCT.
BACKGROUND: Chronic granulomatous disease (CGD) is a primary immunodeficiency characterized by serious infections and inflammation. It can be managed conservatively with prophylactic antimicrobial agents or curatively with hematopoietic stem cell transplantation (HSCT). In the United Kingdom and Ireland there are cohorts of children managed both conservatively and curatively. OBJECTIVES: This study aimed to compare clinical outcomes (mortality and morbidity) in children managed conservatively and curatively. METHODS: Children were identified from specialist centers and advertising through special interest groups. Clinical data were collected from medical records regarding infections, inflammatory complications and growth, other admissions, and curative treatment. Comparisons were made for patients not undergoing HSCT and patients after HSCT. RESULTS: Seventy-three living children were identified, 59 (80%) of whom were recruited. Five deceased children were also identified. Clinical information was available for 62 children (4 deceased). Thirty (48%) children had undergone HSCT. Children who did not undergo transplantation had 0.71 episodes of infection/admission/surgery per CGD life year (95% CI, 0.69-0.75 events per year). Post-HSCT children had 0.15 episodes of infection/admission/surgery per transplant year (95% CI, 0.09-0.21 events per year). The mean z score for height and body mass index (BMI) for age was significantly better in post-HSCT children. Survival in the non-HSCT group was 90% at age 15 years. Survival in the post-HSCT group was 90%. CONCLUSIONS: Children with CGD not undergoing transplantation have more serious infections, episodes of surgery, and admissions compared with post-HSCT children. Children undergoing transplantation have better height for age. Survival is good at the end of the pediatric age range and also after HSCT.
Authors: Alexandra C Battersby; Alexander J Martin; Jessica Tarn; Fai W Ng; Catherine M Cale; David Goldblatt; Andrew R Gennery Journal: J Clin Immunol Date: 2017-03-23 Impact factor: 8.317
Authors: M Hoenig; T Niehues; K Siepermann; E-M Jacobsen; C Schütz; I Furlan; G Dückers; G Lahr; M Wiesneth; K-M Debatin; W Friedrich; A Schulz Journal: Bone Marrow Transplant Date: 2014-06-23 Impact factor: 5.483
Authors: Mark Parta; Corin Kelly; Nana Kwatemaa; Narda Theobald; Diane Hilligoss; Jing Qin; Douglas B Kuhns; Christa Zerbe; Steven M Holland; Harry Malech; Elizabeth M Kang Journal: J Clin Immunol Date: 2017-07-28 Impact factor: 8.317
Authors: M Teresa de la Morena; David Leonard; Troy R Torgerson; Otavio Cabral-Marques; Mary Slatter; Asghar Aghamohammadi; Sharat Chandra; Luis Murguia-Favela; Francisco A Bonilla; Maria Kanariou; Rongras Damrongwatanasuk; Caroline Y Kuo; Christopher C Dvorak; Isabelle Meyts; Karin Chen; Lisa Kobrynski; Neena Kapoor; Darko Richter; Daniela DiGiovanni; Fatima Dhalla; Evangelia Farmaki; Carsten Speckmann; Teresa Español; Anna Shcherbina; Imelda Celine Hanson; Jiri Litzman; John M Routes; Melanie Wong; Ramsay Fuleihan; Suranjith L Seneviratne; Trudy N Small; Ales Janda; Liliana Bezrodnik; Reinhard Seger; Andrea Gomez Raccio; J David M Edgar; Janet Chou; Jordan K Abbott; Joris van Montfrans; Luis Ignacio González-Granado; Nancy Bunin; Necil Kutukculer; Paul Gray; Gisela Seminario; Srdjan Pasic; Victor Aquino; Christian Wysocki; Hassan Abolhassani; Morna Dorsey; Charlotte Cunningham-Rundles; Alan P Knutsen; John Sleasman; Beatriz Tavares Costa Carvalho; Antonio Condino-Neto; Eyal Grunebaum; Helen Chapel; Hans D Ochs; Alexandra Filipovich; Mort Cowan; Andrew Gennery; Andrew Cant; Luigi D Notarangelo; Chaim M Roifman Journal: J Allergy Clin Immunol Date: 2016-09-30 Impact factor: 10.793