Peer providers and linkage with buprenorphine care after hospitalization: A retrospective cohort study.

Background: People with opioid use disorder (OUD) are increasingly started on buprenorphine in the hospital, yet many patients do not attend outpatient buprenorphine care after discharge. Peer providers, people in recovery themselves, are a growing part of addiction care. We examine whether patients who received a low-intensity, peer-delivered intervention during hospitalization had a greater rate of linking with outpatient buprenorphine care relative to those not seen by a peer. Methods: This was a retrospective cohort study of adults with OUD who were started on buprenorphine during hospitalization. The primary outcome was receipt of a buprenorphine prescription within 30 days of discharge. Secondary outcomes included attendance at a follow-up visit with a buprenorphine provider within 30 days and hospital readmission within 90 days. Modified Poisson regression analyses tested for differences in the rate ratios (RR) of each binary outcome for patients who were versus were not seen by a peer provider. Peer notes in the electronic health record were reviewed to characterize peer activities. Results: 111 patients met the study inclusion criteria, 31.5% of whom saw a peer provider. 55.0% received a buprenorphine prescription within 30 days of hospital discharge. Patients with versus without peer provider encounters did not significantly differ in the rates of receiving a buprenorphine prescription (RR = 1.06, 95% CI: 0.74-1.51), hospital readmission (RR = 1.45, 95% CI: 0.80-2.64), or attendance at a buprenorphine follow-up visit (RR = 1.03, 95% CI: 0.68-1.57). Peers most often listened to or shared experiences with patients (68.6% of encounters) and helped facilitate medical care (60.0% of encounters). Conclusions: There were no differences in multiple measures of buprenorphine follow-up between patients who received this low-intensity peer intervention and those who did not. There is need to investigate what elements of peer provider programs contribute to patient outcomes and what outcomes should be assessed when evaluating peer programs.

Staff Attitudes toward Buprenorphine before and after Implementation of an Office-Based Opioid Treatment Program in an Urban Teaching Clinic.

PURPOSE: Improving access to buprenorphine treatment is necessary to address the national opioid use disorder (OUD) crisis. This study investigates attitudes about buprenorphine prescribing among staff at a primary care clinic and compares attitudes before and after implementation of an office-based opioid treatment (OBOT) program. METHODS: Providers and staff in an academic primary care clinic were surveyed prior to and one year following implementation of an OBOT program. Descriptive statistics, Pearson's Chi-2 tests and logistic regression models were used to compare staff and provider attitudes about use of buprenorphine for OUD and to compare attitudes before and after OBOT implementation. RESULTS: At baseline, 20% of staff indicated strong belief that buprenorphine is an effective treatment for OUD and 16% indicated strong belief that primary care providers should prescribe it. Staff appeared less likely than providers to believe strongly that buprenorphine is effective (OR 0.24, 95% CI= 0.08-.78, p = 0.02; aOR 0.28, 95% CI=.08-1.0, p = 0.05 adjusted for age, race and gender). Following implementation of an OBOT program, the percentage of staff who believed strongly in the effectiveness of buprenorphine for OUD increased from 20% to 40% (p = 0.31), and the percentage who believed that primary care providers (PCPs) should prescribe it increased from 16% to 30% (p = 0.52). CONCLUSIONS: Staff in a primary care clinic were less likely than providers to believe in the effectiveness of buprenorphine treatment or that PCPs should prescribe it for OUD. That their beliefs substantially changed after implementation of an OBOT program suggests that direct experience impacts attitudes.

"It's that longitudinal relationship that pays off": A qualitative study of internal medicine residents' perspectives on learning to prescribe buprenorphine.

Background: The opioid use disorder (OUD) epidemic is a national public health crisis. Access to effective treatment with buprenorphine is limited, in part because few physicians are trained to prescribe it. Little is known about how post-graduate trainees learn to prescribe buprenorphine or how to optimally train them to prescribe. We therefore aimed to explore the experiences and attitudes of residents learning to prescribe buprenorphine within two primary care-based opioid treatment models. Methods: We performed semi-structured interviews with second- and third-year internal medicine residents at an urban academic residency program. Participating residents practiced in clinics providing buprenorphine care using either a nurse care manager model or a provider-centric model. Subjects were sampled purposively to ensure that a diversity of perspectives were included. Interviews were conducted until theoretical saturation was reached and were analyzed using principles of thematic analysis. The research team developed a consensus code list. Each transcript was then independently coded by two researchers. The team then summarized each code and generated a set of themes that captured the main ideas emerging from the data. Results: We completed 14 interviews. Participants reported learning to prescribe buprenorphine through didactics, longitudinal outpatient prescribing, mentorship, and inpatient experiences. We characterized their attitudes toward patients with OUD, medication treatment of OUD, their own role in buprenorphine care, and future prescribing. Participants practicing in both clinical models viewed learning to prescribe buprenorphine as a normal part of their training and demonstrated positive attitudes toward buprenorphine prescribing. Conclusions: Longitudinal outpatient experiences with buprenorphine prescribing can prepare residents to prescribe buprenorphine and stimulate interest in prescribing after residency. Both nurse care manager and provider-centric clinical models can provide meaningful experiences for medical residents. Educators should attend to the volume of patients and inductions managed by each trainee, patient-provider continuity, and supporting trainees in the clinical encounter.

Patients Evaluate Visit Notes Written by Their Clinicians: a Mixed Methods Investigation.

BACKGROUND: Patients actively involved in their care demonstrate better health outcomes. Using secure internet portals, clinicians are increasingly offering patients access to their narrative visit notes (open notes), but we know little about their understanding of notes written by clinicians. OBJECTIVE: We examined patients' views on the clarity, accuracy, and thoroughness of notes, their suggestions for improvement, and associations between their perceptions and willingness to recommend clinicians to others. DESIGN: We conducted an online survey of patients in 3 large health systems, June-October 2017. We performed a mixed methods analysis of survey responses regarding a self-selected note. PARTICIPANTS: Respondents were 21,664 patients aged 18 years or older who had read at least 1 open note in the previous 12 months. MAIN MEASURES: We asked to what degree the patient recalled understanding the note, whether it described the visit accurately, whether anything important was missing, for suggestions to improve the note, and whether they would recommend the authoring clinician to others. KEY RESULTS: Nearly all patients (96%) reported they understood all or nearly all of the self-selected note, with few differences by clinician type or specialty. Overall, 93% agreed or somewhat agreed the note accurately described the visit, and 6% reported something important missing from the note. The most common suggestions for improvement related to structure and content, jargon, and accuracy. Patients who reported understanding only some or very little of the note, or found inaccuracies or omissions, were much less likely to recommend the clinician to family and friends. CONCLUSIONS: Patients overwhelmingly report understanding their visit notes and usually find them accurate, with few disparities according to sociodemographic or health characteristics. They have many suggestions for improving their quality, and if they understand a note poorly or find inaccuracies, they often have less confidence in their clinicians.

Mortality After Discontinuation of Primary Care-Based Chronic Opioid Therapy for Pain: a Retrospective Cohort Study.

BACKGROUND: Despite known risks of using chronic opioid therapy (COT) for pain, the risks of discontinuation of COT are largely uncharacterized. OBJECTIVE: To evaluate mortality, prescription opioid use, and primary care utilization of patients discontinued from COT, compared with patients maintained on opioids. DESIGN: Retrospective cohort study of patients with chronic pain enrolled in an opioid registry as of May 2010. PARTICIPANTS: Patients with chronic pain enrolled in the opioid registry of a primary care clinic at an urban safety-net hospital in Seattle, WA. MAIN OUTCOMES AND MEASURES: Discontinuation from the opioid registry was the exposure of interest. Pre-specified main outcomes included mortality, prescription and primary care utilization data, and reasons for discontinuation. Data was collected through March 2015. KEY RESULTS: The study cohort comprised 572 patients with a mean age of 54.9 ± 10.1 years. COT was discontinued in 344 patients (60.1%); 254 (73.8%) discontinued patients subsequently filled at least one opioid prescription in Washington State, and 187 (54.4%) continued to visit the clinic. During the study period, 119 (20.8%) registry patients died, and 21 (3.7%) died of definite or possible overdose: 17 (4.9%) discontinued patients died of overdose, whereas 4 (1.75%) retained patients died of overdose. Most patients had at least one provider-initiated reason for COT discontinuation. Discontinuation of COT was associated with a hazard ratio for death of 1.35 (95% CI, 0.92 to 1.98, p = 0.122) and for overdose death of 2.94 (1.01-8.61, p = 0.049), after adjusting for age and race. CONCLUSIONS: In this cohort of patients prescribed COT for chronic pain, mortality was high. Discontinuation of COT did not reduce risk of death and was associated with increased risk of overdose death. Improved clinical strategies, including multimodal pain management and treatment of opioid use disorder, may be needed for this high-risk group.

Interest in prescribing buprenorphine among resident and attending physicians at an urban teaching clinic.

Background: Opioid use disorders are a major medical and public health concern. Buprenorphine is approved for the treatment of opioid use disorders; however, a shortage of physicians prescribing buprenorphine is a significant barrier to treatment access. The aims of this study were to evaluate opinions of internal medicine attending and resident physicians about buprenorphine and assess interest in becoming waivered to prescribe. Methods: Internal medicine resident and attending physicians at a primary care clinic in a large academic hospital were invited to complete surveys. The study sample was composed of physicians who were not waivered to prescribe buprenorphine. Survey data included demographic information, level of training, buprenorphine waiver status, interest in becoming waivered to prescribe buprenorphine, and beliefs about buprenorphine for treatment of opioid use disorders. High interest in becoming waivered was defined as a Likert response >3 (1 = No interest, 5 = Very interested). Results: Of the 44 physician respondents, 39 were not waivered to prescribe buprenorphine and constituted the sample; of those, 27 were residents and 12 were attending physicians. Twenty-six of the 39 nonwaivered respondents (66.7%) had high interest in becoming waivered. Those with high interest in becoming waivered were significantly more likely to be younger (P = .007) and to strongly believe in buprenorphine effectiveness (P = .023). Discussion: Most physicians in this academic training program showed high interest in prescribing buprenorphine, and belief in buprenorphine effectiveness was associated with high interest in becoming waivered.

Perceptions of Primary Care Notes by Patients With Mental Health Diagnoses.

There are concerns regarding whether patients with mental illness should be provided with access to their electronic medical records. This study compared perceptions of patients with (n = 400) and without (n = 2,134) a mental health diagnosis regarding access to primary care clinic notes through secure online portals. Eligible participants viewed at least 1 clinic note during a 12-month period. Administrative data were used to stratify patients by mental health diagnosis. As we hypothesized, patients with and without mental health diagnoses had similar perceptions about online access to notes.

Patients' perceptions of their doctors' notes and after-visit summaries: A mixed methods study of patients at safety-net clinics.

BACKGROUND: Patients are increasingly offered electronic access to their doctors' notes, and many consistently receive paper After-Visit Summaries. Specific feedback from patients about notes and summaries are lacking, particularly within safety-net settings. DESIGN: A mixed methods study SETTING AND PARTICIPANTS: Patients with poorly controlled diabetes attending two urban safety-net primary care clinics in Washington State. METHODS: Patients read their own most recent clinic note and After-Visit Summary, then completed a brief survey followed by a focus group discussion (3 groups in a large general medicine teaching clinic and 1 in an HIV/AIDS clinic) about their perceptions of the clinic note and After-Visit Summary. RESULTS: Twenty-seven patients participated; 70% were male, 41% were Black, 48% were unemployed or disabled, 56% reported fair/poor health, and 37% had accessed the electronic patient portal. A majority of patients felt their note content was useful (89%); a minority reported that their notes were not accurate (19%), had too much medical jargon (29%), or were too long (26%). Themes identified from the discussions included reliance on the provider to explain confusing content; a desire for more rather than less detail; and perceived inaccuracies, particularly in heavily templated notes. In each focus group, one or more portal users were enthusiastically willing to teach other patients. CONCLUSIONS: The majority of focus group participants at this safety-net site had not accessed the electronic patient portal, but those who had were willing to promote the portal benefits and assist others. Patients identified specific opportunities to improve clinic notes and After-Visit Summaries.

Transitional care clinics for follow-up and primary care linkage for patients discharged from the ED.

OBJECTIVES: Transitional care clinics (TCCs) represent one strategy to facilitate follow-up and primary care linkage for patients with no regular source of care who are discharged from the emergency department (ED). We assessed factors associated with completion of TCC follow-up among these patients and characterized their subsequent ED use. METHODS: Retrospective study of 660 randomly sampled patients with a scheduled appointment to a TCC at time of ED discharge. Patient- and visit-level characteristics were abstracted from the medical records of these patients and linked to a state visit database to characterize ED use after referral. Multiple logistic regression was used to determine factors associated with completion of follow-up and subsequent ED utilization. RESULTS: Half (50%) of the patients completed their follow-up appointment with a mean follow-up time of 6.9days. Non-English language (odds ratio [OR], 2.21; confidence interval [CI], 1.30-3.75) was the only factor associated with improved follow-up; however, patients who were homeless (OR, 0.42; CI, 0.26-0.66) had a substance use history (OR, 0.68; CI, 0.45-1.00), and those with more baseline ED visits (OR, 0.94 per additional ED visit; CI, 0.89-0.99) were significantly less likely to complete follow-up. After adjusting for demographic, clinical, and visit-level characteristics, patients who completed their appointment had significantly fewer ED visits in the subsequent year compared to patients who did not complete their appointment (mean, 2.3 vs 3.3 visits; difference, -1.0 visits in subsequent calendar year; CI for difference, -1.2 to -0.7). CONCLUSION: Transitional care clinics represent a promising strategy to improve the continuity of care for emergency patients and may reduce unnecessary ED use.

Cardiac manifestations and outcomes of COVID-19 vaccine-associated myocarditis in the young in the USA: longitudinal results from the Myocarditis After COVID Vaccination (MACiV) multicenter study.

Background: We aimed to study the clinical characteristics, myocardial injury, and longitudinal outcomes of COVID-19 vaccine-associated myocarditis (C-VAM). Methods: In this longitudinal retrospective observational cohort multicenter study across 38 hospitals in the United States, 333 patients with C-VAM were compared with 100 patients with multisystem inflammatory syndrome in children (MIS-C). We included patients ≤30 years of age with a clinical diagnosis of acute myocarditis after COVID-19 vaccination based on clinical presentation, abnormal biomarkers and/or cardiovascular imaging findings. Demographics, past medical history, hospital course, biochemistry results, cardiovascular imaging, and follow-up information from April 2021 to November 2022 were collected. The primary outcome was presence of myocardial injury as evidenced by late gadolinium enhancement (LGE) on cardiac magnetic resonance (CMR) imaging. Findings: Patients with C-VAM were predominantly white (67%) adolescent males (91%, 15.7 ± 2.8 years). Their initial clinical course was more likely to be mild (80% vs. 23%, p < 0.001) and cardiac dysfunction was less common (17% vs. 68%, p < 0.0001), compared to MIS-C. In contrast, LGE on CMR was more prevalent in C-VAM (82% vs. 16%, p < 0.001). The probability of LGE was higher in males (OR 3.28 [95% CI: 0.99, 10.6, p = 0.052]), in older patients (>15 years, OR 2.74 [95% CI: 1.28, 5.83, p = 0.009]) and when C-VAM occurred after the first or second dose as compared to the third dose of mRNA vaccine. Mid-term clinical outcomes of C-VAM at a median follow-up of 178 days (IQR 114-285 days) were reassuring. No cardiac deaths or heart transplantations were reported until the time of submission of this report. LGE persisted in 60% of the patients at follow up. Interpretation: Myocardial injury at initial presentation and its persistence at follow up, despite a mild initial course and favorable mid-term clinical outcome, warrants continued clinical surveillance and long-term studies in affected patients with C-VAM. Funding: The U.S. Food and Drug Administration.

Reduced toxicity conditioning and allogeneic stem cell transplantation in adults using fludarabine, carmustine, melphalan, and antithymocyte globulin: outcomes depend on disease risk index but not age, comorbidity score, donor type, or human leukocyte antigen mismatch.

Although reduced-intensity conditioning has become standard of care for patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation (HCT), the optimum regimen has yet to be defined, and may depend on pretransplantation patient- and/or disease-specific risk factors. We report here results in 100 adults, ages 18 to 69, with high-risk hematologic malignancy who received conditioning with fludarabine, carmustine, melphalan, and rabbit antithymocyte globulin (FBM-A). Outcomes were stratified using the disease risk index (DRI) as published by Armand et al. (Blood 2012;120:905-913). Median age was 56, and patients were ineligible for standard myeloablative conditioning because of age, organ dysfunction, or prior autologous HCT. Patients underwent transplantation for myeloid (acute myelogenous leukemia, n = 40; myelodysplastic syndrome, n = 24; myelofibrosis, n = 13; other myeloid, n = 2) or lymphoid (acute lymphoblastic leukemia, n = 8; non-Hodgkin lymphoma, n = 8; Hodgkin lymphoma, n = 4, chronic lymphocytic leukemia, n = 1) malignancy. Donors were related in 26 patients (22 matched, 4 mismatched at 1 antigen) and unrelated in 74 (mismatched at 1 or 2 HLA loci in 33); grafts were peripheral blood stem cells in 97 patients, bone marrow in 2, and double cord in 1. According to the DRI, 68 patients were classified as low (n = 1) or intermediate risk (n = 67), and 32 were classified as high (n = 28) or very high risk (n = 4). With a median follow-up of surviving patients of 18 months, the Kaplan-Meier estimate of overall survival at 2 years for patients in the low/intermediate risk group is 80%, compared with 66% in the high/very high group (P = .11). Two-year cumulative incidence of relapse and nonrelapse mortality in the low/intermediate group are 9.9% and 15%, versus 25% and 19% in the high/very high group (respective P values .07 and .81). The cumulative incidence of acute graft-versus-host (GVHD) grades III to IV at 100 days was 8.1%, and the incidence of National Institutes of Health-defined moderate to severe chronic GVHD was 22% at 2 years. No deaths were attributable to chronic GVHD. Survival was not influenced by age, hematopoietic comorbidity index score, donor type, donor gender, or presence of mismatch. We conclude that FBM-A is an effective and safe conditioning regimen for adults up to age 69 with hematologic malignancies who have low-, intermediate-, or high-risk scores according to the DRI.

Implementation Facilitation to Promote Emergency Department-Initiated Buprenorphine for Opioid Use Disorder.

Importance: Emergency department (ED)-initiated buprenorphine for the treatment of opioid use disorder (OUD) is underused. Objective: To evaluate whether provision of ED-initiated buprenorphine with referral for OUD increased after implementation facilitation (IF), an educational and implementation strategy. Design, Setting, and Participants: This multisite hybrid type 3 effectiveness-implementation nonrandomized trial compared grand rounds with IF, with pre-post 12-month baseline and IF evaluation periods, at 4 academic EDs. The study was conducted from April 1, 2017, to November 30, 2020. Participants were ED and community clinicians treating patients with OUD and observational cohorts of ED patients with untreated OUD. Data were analyzed from July 16, 2021, to July 14, 2022. Exposure: A 60-minute in-person grand rounds was compared with IF, a multicomponent facilitation strategy that engaged local champions, developed protocols, and provided learning collaboratives and performance feedback. Main Outcomes and Measures: The primary outcomes were the rate of patients in the observational cohorts who received ED-initiated buprenorphine with referral for OUD treatment (primary implementation outcome) and the rate of patients engaged in OUD treatment at 30 days after enrollment (effectiveness outcome). Additional implementation outcomes included the numbers of ED clinicians with an X-waiver to prescribe buprenorphine and ED visits with buprenorphine administered or prescribed and naloxone dispensed or prescribed. Results: A total of 394 patients were enrolled during the baseline evaluation period and 362 patients were enrolled during the IF evaluation period across all sites, for a total of 756 patients (540 [71.4%] male; mean [SD] age, 39.3 [11.7] years), with 223 Black patients (29.5%) and 394 White patients (52.1%). The cohort included 420 patients (55.6%) who were unemployed, and 431 patients (57.0%) reported unstable housing. Two patients (0.5%) received ED-initiated buprenorphine during the baseline period, compared with 53 patients (14.6%) during the IF evaluation period (P < .001). Forty patients (10.2%) were engaged with OUD treatment during the baseline period, compared with 59 patients (16.3%) during the IF evaluation period (P = .01). Patients in the IF evaluation period who received ED-initiated buprenorphine were more likely to be in treatment at 30 days (19 of 53 patients [35.8%]) than those who did not 40 of 309 patients (12.9%; P < .001). Additionally, there were increases in the numbers of ED clinicians with an X-waiver (from 11 to 196 clinicians) and ED visits with provision of buprenorphine (from 259 to 1256 visits) and naloxone (from 535 to 1091 visits). Conclusions and Relevance: In this multicenter effectiveness-implementation nonrandomized trial, rates of ED-initiated buprenorphine and engagement in OUD treatment were higher in the IF period, especially among patients who received ED-initiated buprenorphine. Trial Registration: ClinicalTrials.gov Identifier: NCT03023930.

Long-term outcome of patients with metastatic breast cancer treated with high-dose chemotherapy and transplantation of purified autologous hematopoietic stem cells.

Metastatic breast cancer remains a major treatment challenge. The use of high-dose chemotherapy (HDCT) with rescue by autologous mobilized peripheral blood (MPB) is controversial, in part because of contamination of MPB by circulating tumor cells. CD34(+)Thy-1(+) selected hematopoietic stem cells (HSC) represent a graft source with a greater than 250,000-fold reduction in cancer cells. Here, we present the long-term outcome of a pilot study to determine feasibility and engraftment using HDCT and purified HSC in patients with metastatic breast cancer. Twenty-two patients who had been treated with standard chemotherapy were enrolled into a phase I/II trial between December 1996 and February 1998, and underwent HDCT followed by rescue with CD34(+)Thy-1(+) HSC isolated from autologous MPB. More than 12 years after the end of the study, 23% (5 of 22) of HSC recipients are alive, and 18% (4 of 22) are free of recurrence with normal hematopoietic function. Median progression-free survival (PFS) was 16 months, and median overall survival (OS) was 60 months. Retrospective comparison with 74 patients transplanted between February 1995 and June 1999 with the identical HDCT regimen but rescue with unmanipulated MPB indicated that 9% of patients are alive, and 7% are without disease. Median PFS was 10 months, and median OS was 28 months. In conclusion, cancer-depleted HSC following HDCT resulted in better than expected 12- to 14-year PFS and OS in a cohort of metastatic breast cancer patients. These data prompt us to look once again at purified HSC transplantation in a protocol powered to test for efficacy in advanced-stage breast cancer patients.

Low Dose Buprenorphine Induction With Full Agonist Overlap in Hospitalized Patients With Opioid Use Disorder: A Retrospective Cohort Study.

OBJECTIVE: To describe the outcomes of buprenorphine/naloxone low dose induction with overlap of full opioid agonists among hospitalized patients with opioid use disorder (OUD) as an alternative to standard induction strategies. METHODS: Retrospective cohort study of patients with OUD who were admitted to the hospital over a 1-year period and initiated ono buprenorphine using initial doses of 0.5 mg and gradually increased while the patient remained on full agonists. Descriptive variables included basic demographics, reason for switching to buprenorphine, baseline opioid and morphine equivalent dose. The primary outcome was a successful transition defined by the patient leaving the hospital with a buprenorphine prescription. Bivariate analysis identified factors associated with unsuccessful medication transitions. Secondary outcomes included reported withdrawal symptoms and 30 day follow up to an outpatient buprenorphine program. RESULTS: Sixty two patients underwent low dose with overlap induction during the study period. Fourteen patients were on methadone for OUD before hospital admission. Fifty one patients (82%) successfully left the hospital with a prescription for buprenorphine. Factors associated with lower likelihood of success included older age, transitioning due to discharge placement needs and presence of withdrawal symptoms during the transition. Overall, 66% (N = 23) of patients referred within the same health care system followed up within 30 days. CONCLUSIONS: Low dose inductions with overlap of full opioid agonists were largely successful in transitioning hospitalized patients from full agonist opioids to buprenorphine. However, there were several factors associated with lower likelihood of success. Future work could focus on treatment of withdrawal symptoms and system-level changes ensuring patient-centered medication decisions.

Optimizing evaluation in pediatric and young adult patients with Marfan syndrome: Novel longitudinal metrics to track growth of aortic structures.

OBJECTIVE: Surveillance metrics in pediatric and young adult Marfan syndrome (pMFS) are challenging. We evaluated the utility of aortic root cross-sectional area/height index (CSA/Ht) on echocardiogram among pMFS patients as a risk stratification and surgical triage metric. METHODS: Genotype or phenotype positive pMFS patients aged 25 years or younger seen at our center from 2001 to 2020 were identified. Time-related transition to surgery was modeled using parametric methods. Predictive utility of CSA/Ht compared with aortic root diameter (ARd) and root Z score (ARz) were modeled using nonlinear multivariable parametric and nonparametric longitudinal regression models. RESULTS: Seventy-nine patients (43% female) presented at median age of 5.8 years (15th-85th percentile, 0.75-17 years) with median follow-up of 4.4 years (range, 0-18.5 years). Baseline echocardiography data were: CSA/Ht, 3.9 ± 1.4 cm2/m; ARd, 2.4 ± 0.89 cm; and ARz, 2.4 ± 1.7. CSA/Ht tracked ARd better compared with ARz (r = 0.91 vs 0.24). Eighteen patients underwent surgery. Surgical procedures included at least 2 components in 17 (aortic, mitral, tricuspid, aortic root, and arch procedures) and isolated mitral valve procedures in 1 patient. Time-related transition to surgery showed a prominent early phase to 1 year post presentation, followed by a slowly increasing late phase. CSA/Ht had a more linear correlation versus ARz during periods of rapid somatic growth in surgical patients. Surgical repair occurred at CSA/Ht between 5 and 7 cm2/m. CONCLUSIONS: CSA/Ht tracked ARd well over time. CSA/Ht between 5 and 7 cm2/m might be a promising metric for surgical triage in pMFS patients. CSA/Ht surgical threshold values in pMFS patients occurred at lower than current accepted "surgical" threshold values for CSA/Ht in adult Marfan syndrome patients.