Hyperhemolysis is a life-threatening condition of exaggerated hemolysis of red blood cells which occurs in patients receiving chronic transfusion therapy. We present a 19-year-old male with the β-thalassemia major with an episode of hyperhemolysis. Hemolysis was initially unresponsive to immunosuppression but responded after the addition of eculizumab. Several weeks after stabilization, hemolysis returned; which was also managed with immunosuppression and eculizumab. Hyperhemolysis presents significant challenges in β-thalassemia due to the underlying dysfunctional erythropoiesis and transfusion dependence. Aggressive immunosuppression combined with eculizumab successfully slowed the hemolysis and allowed for the resumption of transfusions.
Objective Chemotherapy-induced nausea and vomiting (CINV) is characterized by disabling nausea and emesis that can recur throughout the treatment of cancer and affects approximately 59% of pediatric and young adult patients. CINV can be associated with significant clinical morbidity, frequent hospital admissions, negative effects on health care related quality-of-life and can exhibit downstream effects such as weight loss that can worsen overall outcomes. Despite the vast number and potential combinations of pharmacotherapies and lifestyle modifications available to manage CINV in children, there are currently no clinical action tools offered to manage this condition better at home. We aimed to develop and assess an evidence-based, personalized pictogram-based nausea action plan (NAP) to aid providers, parents, and patients in the management of CINV. Methods The USNAP (Figure 1) facilitates the management of CINV by using a health literacy-informed approach to provide instructions for pharmacotherapies and lifestyle modifications. This study included Part 1 (Pictogram Validation) and Part 2 (Assessment). For Part 1, Pictogram transparency, translucency, and recall were assessed by parent survey with transparency ≥85%, mean translucency score ≥5, recall ≥85% required for validation. For Part 2, the USNAP was assessed by parents, clinical librarians, and clinicians. Patient/caregiver perceptions (n=27) were assessed using the Consumer Information Rating Form (17 questions) to gauge comprehension, design quality and usefulness. Readability was assessed by 5 formulas and a Readability Consensus Score was calculated. Clinical Librarians (n=2) used the Patient Education Materials Assessment Tool to measure the understandability (19 questions) and actionability (7 questions) of the plan and audiovisual educational content (>80% acceptable.) Suitability was assessed by clinicians (n=16) using Doaks' Suitability Assessment of Materials (superior≥70% rating). Results All 15 pictograms demonstrated appropriate transparency, translucency, and recall. Patient/caregiver perceptions reflected appropriate comprehension, design quality, and usefulness. The Readability Composite Score measured at a fourth-grade level. Clinical librarians reported acceptable understandability and actionability. Clinicians reported superior suitability. Conclusion The Uniformed Services Nausea Action Plan (USNAP) is the first clinical action tool designed to assist in managing CINV at home. Although recent clinical practice updates provide guidance on the therapeutic management of CINV in pediatric and young adult patients with cancer, these recommendations do not fully address the needs of the patient at home or adequately inform in instances of low health literacy. The USNAP seeks to mitigate this by making the clinical practice guidance useful to patients and caregivers at home. In addition, the USNAP is poised to identify other urgent clinical developments for patients with cancer that could masquerade as nausea and may serve as an early warning sign in these instances. The USNAP met all criteria for clinical implementation. The USNAP has potential to become an important tool in the care of patients with CINV, improving both quality-of-care and clinical outcomes. Future study of USNAP implementation for treating children with chronic CINV is needed. Figure 1 Figure 1. Disclosures No relevant conflicts of interest to declare.
This a preprint and has not been peer reviewed. Data may be preliminary.
Background: Second malignant neoplasms (SMN) are among the most serious long-term adverse health conditions in cancer survivors. The aim of this study was to characterize clinical findings of patients who developed thyroid cancers as SMN, and to examine genomic alterations in thyroid cancer tissue. Methods: Retrospective analysis of medical records from patients seen for management of thyroid cancer over 10-year period was performed. Clinical and pathologic data were retrieved from their medical charts. Tumor DNA and RNA were extracted from formalin-fixed, paraffin-embedded tissue and subjected to next-generation sequencing (NGS) using Ion Torrent Oncomine Focus Assay. Microfluidic digital polymerase chain reactions (PCRs) were performed using QIAcuity Digital PCR System to identify BRAFV600E mutations and RET/PTC fusions. Results: Sixteen of 620 patients operated for thyroid cancer had history of previously diagnosed malignancy. Eight patients were male and eight patients were female, with a median age at diagnosis of 58.5 years (range, 4–78). Four patients had history of pediatric malignancy (PedCa), and 12 patients had a history of prior malignancy as an adult (AdCa). The latency periods for development of SMN in PedCa and AdCa patients were 10.8 (±5.2) years and 9.5 (±5.2) years, respectively. Histopathology revealed papillary thyroid cancers in 15 cases, and follicular thyroid cancer in 1 case. All tumors were classified as T1 or T2, and there were no patients presenting with metastases at the time of surgery. Genomic alterations were detected in 13/16 (81.2%) tumors including eight gene mutations ( BRAFV600E (N = 4), RAS (N = 2), PI3CA (N = 2) and five gene fusions ( RET/PTC1 (N = 4) and STRN/ALK (N = 1). In patients with PedCa and AdCa, mutations were detected in 1/4 (25%) and 7/12 (58.3%), respectively, p = 0.56; and fusions were detected in 3/4 (75%) and 2/12 (16.6%), respectively, p = 0.06. In patients with and without history of therapeutic irradiation, mutations were detected with the same frequencies (5/10 (50%), and 3/6 (50%), respectively, p = 1.0). Gene fusions were detected in patients with and without history of irradiation in 5/10 (55.5%) and 0/6 (0%), respectively, p = 0.09. Conclusions: Monitoring of cancer survivors for thyroid disorders allowed diagnosis of second thyroid cancers at early stages. Second thyroid cancers harbor genomic alterations that are typical for sporadic as well as for radio-induced thyroid cancers.
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