ABSTRACT
Introduction: Proactive surveillance by a critical care outreach team (CCOT) can promote early recognition of deterioration in hospitalized patients but is uncommon in pediatric rapid response systems (RRSs). After our children's hospital introduced a CCOT in 2019, we aimed to characterize early implementation outcomes. We hypothesized that CCOT rounding would identify additional children at risk for deterioration. Methods: The CCOT, staffed by a dedicated critical care nurse (RN), respiratory therapist, and attending, conducts daily in-person rounds with charge RNs on medical-surgical units, to screen RRS-identified high-risk patients for deterioration. In this prospective study, observers tracked rounds discussion content, participation, and identification of new high-risk patients. We compared 'identified-patient-discussions' (IPD) about RRS-identified patients, and 'new-patient-discussions' (NPD) about new patients with Fisher's exact test. For new patients, we performed thematic analysis of clinical data to identify deterioration related themes. Results: During 348 unit-rounds over 20 days, we observed 383 discussions - 35 (9%) were NPD. Frequent topics were screening for clinical concerns (374/383, 98%), active clinical concerns (147/383, 39%), and watcher activation (66/383, 17%). Most discussions only included standard participants (353/383, 92%). Compared to IPD, NPD more often addressed active concerns (74.3% vs 34.8%, p < 0.01) and staffing resource concerns (5.7% vs 0.6%, p < 0.04), and more often incorporated extra participants (25.7% vs 6%, p < 0.01). In thematic analysis of 33 new patients, most (29/33, 88%) had features of deterioration. Conclusion: A successfully implemented CCOT enhanced identification of clinical deterioration not captured by existing RRS resources. Future work will investigate its impact on operational safety and patient-centered outcomes.
ABSTRACT
PURPOSE: Deficiency of adenosine deaminase 2 (DADA2) is an inherited inborn error of immunity, characterized by autoinflammation (recurrent fever), vasculopathy (livedo racemosa, polyarteritis nodosa, lacunar ischemic strokes, and intracranial hemorrhages), immunodeficiency, lymphoproliferation, immune cytopenias, and bone marrow failure (BMF). Tumor necrosis factor (TNF-α) blockade is the treatment of choice for the vasculopathy, but often fails to reverse refractory cytopenia. We aimed to study the outcome of hematopoietic cell transplantation (HCT) in patients with DADA2. METHODS: We conducted a retrospective study on the outcome of HCT in patients with DADA2. The primary outcome was overall survival (OS). RESULTS: Thirty DADA2 patients from 12 countries received a total of 38 HCTs. The indications for HCT were BMF, immune cytopenia, malignancy, or immunodeficiency. Median age at HCT was 9 years (range: 2-28 years). The conditioning regimens for the final transplants were myeloablative (n = 20), reduced intensity (n = 8), or non-myeloablative (n = 2). Donors were HLA-matched related (n = 4), HLA-matched unrelated (n = 16), HLA-haploidentical (n = 2), or HLA-mismatched unrelated (n = 8). After a median follow-up of 2 years (range: 0.5-16 years), 2-year OS was 97%, and 2-year GvHD-free relapse-free survival was 73%. The hematological and immunological phenotypes resolved, and there were no new vascular events. Plasma ADA2 enzyme activity normalized in 16/17 patients tested. Six patients required more than one HCT. CONCLUSION: HCT was an effective treatment for DADA2, successfully reversing the refractory cytopenia, as well as the vasculopathy and immunodeficiency. CLINICAL IMPLICATIONS: HCT is a definitive cure for DADA2 with > 95% survival.
