A Multicenter Retrospective Analysis Stressing the Importance of Long-Term Follow-Up after Hematopoietic Cell Transplantation for ß-Thalassemia.

Allogeneic hematopoietic cell transplantation (HCT) is curative in patients with ß-thalassemia major. However, most reports on HCT outcomes lack long-term follow-up data with the exception of single-center reports. An international multicenter retrospective data collection and analysis was conducted in 176 ß-thalassemia patients who were 1 year or beyond after first HCT to evaluate follow-up methods and outcomes at 7 centers. Median age at HCT was 5.5 years (range, .6 to 18.5), and median follow-up was 7 years (range, 1 to 20). HCT was predominantly from HLA-matched related donors (91%) with bone marrow as stem cell source (91%) and myeloablative conditioning regimens (88%). Late mortality or persistent chronic graft-versus-host disease (GVHD) was rare (<2%). Graft rejection was reported in 23% (24% of these occurred beyond 1 year) post-HCT. Of 119 patients with donor chimerism results available for ≥4 years post-HCT, 50% had >95%, 22% had 50% to 95%, 7% had 20% to 50% and 25 (21%) had <20% donor chimerism. Organ dysfunction was identified in 10% pre-HCT and in 20% post-HCT even without complete clinical details on all patients. Hypogonadism and elevated creatinine for age were most commonly reported and significantly higher in recipients ≥ 7 years at the time of HCT (P = .007) and in those with pre-existing morbidity before HCT (P = .02). Outcomes were unaffected by pre-HCT ferritin or GVHD. Mean z scores for height and weight were low at baseline and remained low post-HCT (79%), confirming that growth impairment from disease lacked recovery post-HCT during this follow-up period. HCT for ß-thalassemia has a high rate of cure and low mortality, especially in the young and from HLA-matched related donors. Half of the number of recipients live with mixed chimerism that requires continued follow-up because of a risk of late graft rejection (14%). Organ function after HCT when <7 years of age was generally preserved. Hypogonadism, renal dysfunction, and growth impairment that failed to correct were late complications identified most frequently in older transplant recipients. Systematic follow-up of individual organs such as lung and heart were inadequate but important. These data support the development of simple measures of uniformly tracking long-term HCT outcomes and organ functions in children and adolescents who undergo HCT for thalassemia, allowing for systematic identification and implementation of standardized surveillance strategies and interventions.

Multidisciplinary Management of Tethered Spinal Cord Syndrome in Children: Operationalizing an Outpatient Patient-Centered Workflow.

INTRODUCTION: Multidisciplinary care for patients with tethered spinal cord syndrome (TCS) is valuable in ensuring comprehensive evaluation, timely follow-up, optimal functional outcome, and patient-centered care. The family-centered focus aims to minimize patient and parental burdens associated with care coordination. We present our first-year institutional experience in operationalizing a multidisciplinary, patient-centered, pediatric tethered cord clinic (TCC) to manage routine, long-term surgical follow-up for children with non-myelomeningocele-related tethered spinal cords. METHODS: TCC is composed of three surgical services: orthopedic surgery, neurosurgery, urology. A retrospective chart review of patients seen in the TCC from January 2019 to January 2020 was conducted. Patients enrolled in the clinic were intended for long-term follow-up. Demographic and outcome variables were collected. RESULTS: Fifty-nine patients were seen in TCC. Types of tethered spinal cords amongst these patients were the following: fatty filum (62.7%), dermal sinus tract (15.2%), meningocele manqué (8.4%), lipomyelomeningocele (6.7%), low lying conus medullaris (5.1%), and sacral arachnoid cyst (1.7%). Age at diagnosis was 1.31 ± 2.21 (median: 0.25 years) and at follow-up was 9.0 ± 5.18 years (median: 8 years). A total of 50.9% of patients were female, and 93.2% had a prior untethering procedure. Of all patients, 6.8% have no surgical intervention and continue to be monitored conservatively for evidence of decline. All three services evaluated 84.8% of patients during the same clinic session, while 15.3% of patients were seen by two of the services, and 20.3% of patients were able to schedule related imaging or diagnostic testing during the same visit. CONCLUSION: We describe successful implementation of a multidisciplinary pediatric TCC and document the first year of experience. The TCC streamlines care, decreases burden on families, and reduces those lost to follow-up. Complex disease pathologies, even when clinically stable, require long-term follow-up with multiple subspecialties and benefit from multidisciplinary clinics.

Corrigendum: Scraping the Web for Public Health Gains: Ethical Considerations from a 'Big Data' Research Project on HIV and Incarceration.

[This corrects the article DOI: 10.1093/phe/phaa006.][This corrects the article DOI: 10.1093/phe/phaa006.].

Surveillance of the eye and vision in clinical trials of CP-675,206 for metastatic melanoma.

PURPOSE: To determine the ocular safety of CP-675,206 (Pfizer, New York, New York, USA), a fully human anti-cytotoxic T lymphocyte-associated antigen 4 monoclonal antibody in clinical trials of immunotherapy of metastatic melanoma. DESIGN: Prospective, nonrandomized study of the eye and vision in phase I/II clinical trials of CP-675,206 in metastatic melanoma conducted at the University of California, Los Angeles. METHODS: Patients with regional or distant metastatic melanoma were enrolled in phase I/II clinical trials evaluating the safety and antitumor efficacy of CP-675,206 alone or in combination with melanoma antigen peptide-pulsed dendritic cell vaccines. Ophthalmic evaluation was performed at the onset of CP-675,206 immunotherapy (baseline evaluation), two months or more after the onset of CP-675,206 immunotherapy (end-study evaluation), and at two- to three-month intervals thereafter in patients who continued to receive CP-675,206 immunotherapy (poststudy evaluation). Baseline and end-study evaluations included comprehensive ophthalmic examination, psychophysical and electrophysiologic visual function assessment, fundus photography, fluorescein angiography, and visual function assessment. RESULTS: Twenty patients with metastatic melanoma arising from the skin, mucosa, eye, or unknown site were evaluated. Systemic toxicity attributed to CP-675,206 included dermatologic manifestations, diarrhea, and autoimmune hepatitis with panhypopituitarism. A subset of patients receiving CP-675,206 demonstrated antitumor efficacy with partial response or complete response of metastatic melanoma. Comparison of ophthalmic baseline with end-study evaluations in all 20 patients and limited-term poststudy evaluations showed no adverse effect of CP-675,206 immunotherapy on the eye or vision. CONCLUSIONS: In this study, CP-675,206 immunotherapy for metastatic melanoma did not adversely affect the eye or vision.