Chronic granulomatous disease (CGD) is a primary immunodeficiency resulting in life-threatening infections and inflammatory complications. Allogeneic hematopoietic cell transplantation (allo-HCT) can cure the disease, but the indication to transplant remains controversial. We performed a retrospective multicenter study of 712 patients with CGD who underwent allo-HCT transplantation from March 1993 through December 2018. We studied 635 children (aged 1 antigen mismatch. Choice of conditioning regimen did not influence OS or EFS. In summary, we report an excellent outcome after allo-HCT in CGD, with low incidence of graft failure and mortality in all ages. Older patients and recipients of 1-antigen-mismatched grafts had a less favorable outcome. Transplantation should be strongly considered at a younger age and particularly in the presence of a well-matched donor.

To the Editor The Viewpoint by Mr Adibi and colleagues¹ proposed some novel approaches for evaluating the performance and utility of published CPMs. We agree with the authors that real-time validation of CPMs with data from routine care is a promising alternative to using data collected for specific research questions. With this strategy, the performance of all competing models is continuously monitored, and the model(s) with the best performance will stand out. Importantly, it is almost cost-free and harmless to patients.

Background: Carcinoembryonic antigen (CEA) as a diagnostic or prognostic marker has been widely studied in patients with lung cancer. However, the relationship between serum CEA and tumor metastasis in lung cancer remains controversial. This study aimed to investigate the ability of serum CEA to assess tumor metastasis in lung cancer patients. Methods: We performed a retrospective analysis of 238 patients diagnosed with lung cancer from January to December 2016 at pneumology department of Dazhou Central Hospital (Dazhou, China). Serum CEA levels were quantified in each patient at the time of diagnosis of lung cancer. Metastasis was confirmed by computed tomography (CT), and/or positron emission tomography (PET) and/or surgery or other necessary detecting methods. Results: Of the 213 patients eligible for final analysis, 128 were diagnosed with metastasis and 85 were diagnosed without metastasis. Compared to non-metastatic patients, the serum CEA was markedly higher in patients with metastasis (p < 0.001), and the area under the curve (AUC) was 0.724 (95% CI [0.654–0.793]). Subsequent analyses regarding the number and location of tumor metastases showed that CEA also had clinical value for multiple metastases versus single metastasis (AUC = 0.780, 95% CI [0.699–0.862]) and distant metastasis versus non-distant metastasis (AUC = 0.815, 95% CI [0.733–0.897]). In addition, we found that tumor size, histology diagnosis, age and gender had no impact on the assessment performance of CEA. Conclusion: Our study suggested the serum CEA as a valuable marker for tumor metastases assessment in newly diagnosed lung cancer patients, which could have some implications in clinical application.

Clinicians increasingly rely on prediction models to guide treatment choices. Most prediction models, however, are developed using observational data that include some patients who have already received the treatment the prediction model is meant to inform. Special attention to the causal role of those earlier treatments is required when interpreting the resulting predictions. "Causal blind spots" were identified in 3 common approaches to handling treatment when developing a prediction model: including treatment as a predictor, restricting to persons taking a certain treatment, and ignoring treatment. Through several real examples, this article illustrates how the risks obtained from models developed using such approaches may be misinterpreted and can lead to misinformed decision making. The discussion covers issues attributable to confounding, selection, mediation, and changes in treatment protocols over time. An extension of guidelines for the development, reporting, and evaluation of prediction models is advocated to avoid such misinterpretations. Developers must ensure that the intended target population for the model, and the treatment conditions under which predictions hold, are clearly communicated. When prediction models are intended to inform treatment decisions, they need to provide estimates of risk under the specific treatment (or intervention) options being considered, known as "prediction under interventions." Next to suitable data, this requires causal reasoning and causal inference techniques during model development and evaluation. Being clear about what a given prediction model can and cannot be used for prevents misinformed treatment decisions and thereby prevents potential harm to patients.

We aimed to assess sex differences in treatment outcomes and adherence comparing men who have sex with women (MSW), men who have sex with men (MSM), and women who have sex with men (WSM), as well as men and women who inject drugs living with HIV on combination antiretroviral therapy (ART) in Guangdong, China. We performed a retrospective observational cohort study with data from the National Free Antiretroviral Treatment Program database. We included ART-naive patients aged 18 to 80 years who had contracted HIV through sex or injecting drugs, initiated first-line ART between January 2004 and December 2016, and had at least 60 days of follow-up. Participants were followed for five years. Kaplan-Meier analysis and Cox proportional hazard models were used to evaluate all-cause mortality. Cumulative incidence function and Cox proportional hazards models accounting for competing risks were used to evaluate disease progression to AIDS. Modified Poisson regression models were used to evaluate immunological and virological responses and loss to follow-up. Repeated measures analysis was used to evaluate regular CD4+ cell count, HIV viral load monitoring, ART adherence, side effects, and interruption of ART. We included 26,409 persons living with HIV. 21,779 (82·5%) people acquired HIV through sex (5118 WSM [23·5%], 8506 MSW [39·0%], 8175 MSM [37·5%]), and 4610 people (17·5%) through injection drug use (249 women [5·4%], 4361 men [94·6%]). Among those infected through sex, MSW had increased risks of all-cause mortality (adjusted hazard ratio [aHR] 1·48, 95% CI 1·20-1·83), progression to AIDS (1·27, 1·09-1·47), virological failure (adjusted incidence rates ratio [aIRR] 1·27, 95% CI 1·09-1·48), and loss to follow-up (1·22, 1·10-1·35) compared to WSM. In contrast, MSM had lower risk of all-cause mortality (aHR 0·49, 95%CI 0·32-0·76), disease progression to AIDS (0·83, 0·68-1·00), and virological failure (aIRR 0·78, 95%CI 0·65-0·94), were more likely to receive regular CD4+ cell count (1·08, 1·07-1·10) and HIV viral load monitoring (1·13, 1·12-1·15), were less likely to report missing ART doses (0·54, 0·49-0·61), interrupt ART (0·34, 0·26-0·44), or be lost to follow-up (0·56, 0·49-0·65) compared to WSM. Men who inject drugs were almost twice as likely as women who inject drugs to die (aHR 1·72, 95%CI 1·03-2·85), experience disease progression to AIDS (2·05, 1·18-3·57), virological failure (aIRR 1·81, 95%CI 1·19-2·76), report ART side effects (1·78, 1·43-2·22), and interruptions in ART (2·29, 1·50-3·50). Our findings highlight the importance of identifying potentially at-risk MSW and promoting HIV education and testing among them. Particular attention is warranted among men who inject drugs to improve timely HIV diagnosis, drug interaction management, and retention in treatment. Additional research from rural settings is needed to assess the long-term treatment outcomes and adherence in MSM with HIV.