Dataset Information

Perceptions of patient disease burden and management approaches in systemic mastocytosis: Results of the TouchStone Healthcare Provider Survey.

ABSTRACT:

Background

Systemic mastocytosis (SM) is a rare clonal neoplasm driven by the KIT D816V mutation and has a broad range of debilitating symptoms. In this study, the authors evaluated SM disease perceptions and management strategies among US health care providers (HCPs).

Methods

Hematologist/oncologist (H/O) HCPs and allergist/immunologist (A/I) HCPs who were treating four or more patients with SM completed an online, 51-item TouchStone HCP Survey, which queried provider characteristics, perceptions of disease burden, and current management. Descriptive analyses by specialty and SM subtype were performed.

Results

Of 304 HCPs contacted, 111 (37%) met eligibility criteria, including 51% A/I specialists and 49% H/O specialists. On average, the HCPs had 14 years of practice experience and cared for 20 patients with SM. A/I HCPs saw more patients with nonadvanced SM (78%) compared with H/O HCPs, who saw similar proportions of patients with nonadvanced SM (54%) and advanced SM (46%). HCPs reported testing 75% of patients for the KIT D816V mutation and found an estimated prevalence of 47%. On average, HCPs estimated 8 months between symptom onset and SM diagnosis. HCPs reported that 62% of patients with indolent SM felt depressed or discouraged because of symptoms. In terms of treatment goals for SM, both types of specialists prioritized symptom improvement for nonadvanced SM and improved survival for advanced SM while also prioritizing improving patient quality of life.

Conclusions

Both A/I and H/O specialists highlighted unmet needs for patients with SM. The HCPs surveyed reported a lower rate of KIT D816V mutations and a perceived shorter time between symptom onset and SM diagnosis compared with published estimates.

Lay summary

Specialists treating systemic mastocytosis (SM) completed a 51-item questionnaire about their clinical practices and perceptions of disease impact. The study included 111 hematology, oncology, allergy, and immunology physicians. Physicians reported that most patients had nonadvanced disease, yet SM symptoms significantly disrupted their patients' lives. Physicians estimated that SM is diagnosed within months of symptom onset, in contrast with published reports of years' long delays reported by patients with SM. This study identified unmet needs that can inform educational and patient management priorities in this rare disease.

SUBMITTER: Mesa RA

PROVIDER: S-EPMC9804550 | biostudies-literature | 2022 Oct

REPOSITORIES: biostudies-literature

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Publications

Perceptions of patient disease burden and management approaches in systemic mastocytosis: Results of the TouchStone Healthcare Provider Survey.

Mesa Ruben A RA Sullivan Erin M EM Dubinski David D Carroll Brittany B Slee Valerie M VM Jennings Susan V SV Finnerty Celeste C CC Bohannon Linda S LS Mathias Susan D SD Lahue Betsy J BJ Castells Mariana C MC

Cancer 20220823 20

<h4>Background</h4>Systemic mastocytosis (SM) is a rare clonal neoplasm driven by the KIT D816V mutation and has a broad range of debilitating symptoms. In this study, the authors evaluated SM disease perceptions and management strategies among US health care providers (HCPs).<h4>Methods</h4>Hematologist/oncologist (H/O) HCPs and allergist/immunologist (A/I) HCPs who were treating four or more patients with SM completed an online, 51-item TouchStone HCP Survey, which queried provider characteris ...[more]

PMID: 35996871

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Project description:BackgroundSystemic mastocytosis (SM) is a rare clonal neoplasm driven by KIT D816V and other mutations. Data were collected from the patient perspective on disease burden and included an SM-specific symptom assessment tool.MethodsUS adults aged 18 years and older with a self-reported SM diagnosis completed an online TouchStone SM Patient Survey of 100 items, including the 12-item Short-Form Health Survey, the Indolent Systemic Mastocytosis Symptom Assessment Form, and the Work Productivity and Activity Impairment Questionnaire, as well as questions about SM diagnosis, the impact of SM on daily activities, work impairment, and health care use. The results were analyzed using descriptive statistics.ResultsFifty-six individuals completed the survey (89% women; median age, 48 years; mean time since diagnosis, 6.7 years), reporting indolent SM (66%), aggressive SM (9%), smoldering SM (5%), and unknown SM subtype (18%). Over a 1-year recall, respondents reported seeking emergency care for anaphylaxis (30%) and taking three or more prescription medications (52%) for SM. Over one half of patients (54%) reduced their work hours because of SM, and 64% avoided leaving home because of symptoms. A majority of respondents (93%) had experienced ≥10 SM-related symptoms, noting that the most bothersome were anaphylactic episodes (18%), abdominal/stomach pain (16%), diarrhea/loose stools (13%), and fatigue (11%). Whereas an Indolent Systemic Mastocytosis Symptom Assessment Form-derived total symptom score of 28 is used to indicate moderate-to-severe symptoms, the mean total symptom score was 52.7. Mental and physical component summary scores from the 12-item Short-Form Health Survey were below population norms.ConclusionsPatients who were surveyed reported substantial symptom burden and unmet needs because of SM, as evidenced by seeking emergency care and reporting bothersome symptoms, poor quality of life, and reduced work hours and productivity.Lay summaryThe objective of this research was to understand the burden and unmet needs in the rare disease of systemic mastocytosis (SM) to guide future care. Fifty-six patients completed an online survey containing questions about their diagnosis, medications, health care use, quality of life, and SM symptoms. The results demonstrated that SM is associated with severe and burdensome symptoms, anaphylactic events, emergency department visits, use of multiple medications, reduced ability to work, and poor physical and psychological quality of life. These findings suggest the need for future advances to address unmet needs in patients affected by SM.

Project description:Mastocytosis is a myeloproliferative neoplasm characterized by expansion of abnormal mast cells (MCs) in various tissues, including skin, bone marrow, gastrointestinal tract, liver, spleen, or lymph nodes. Subtypes include indolent systemic mastocytosis, smoldering systemic mastocytosis and advanced systemic mastocytosis (AdvSM), a term collectively used for the three most aggressive forms of the disease: aggressive systemic mastocytosis, mast cell leukemia, and systemic mastocytosis with an associated clonal hematological non-mast cell disease (SM-AHNMD). MC activation and proliferation is physiologically controlled in part through stem cell factor (SCF) binding to its cognate receptor, KIT. Gain-of-function KIT mutations that lead to ligand-independent kinase activation are found in most SM subtypes, and the overwhelming majority of AdvSM patients harbor the KITD816V mutation. Several approved tyrosine kinase inhibitors (TKIs), such as imatinib and nilotinib, have activity against wild-type KIT but lack activity against KITD816V. Midostaurin, a broad spectrum TKI with activity against KITD816V, has a 60% clinical response rate, and is currently the only drug specifically approved for AdvSM. While this agent improves the prognosis of AdvSM patients and provides proof of principle for targeting KITD816V as a driver mutation, most responses are partial and/or not sustained, indicating that more potent and/or specific inhibitors are required. Avapritinib, a KIT and PDGFRα inhibitor, was specifically designed to inhibit KITD816V. Early results from a Phase 1 trial suggest that avapritinib has potent antineoplastic activity in AdvSM, extending to patients who failed midostaurin. Patients exhibited a rapid reduction in both symptoms as well as reductions of bone marrow MCs, serum tryptase, and KITD816V mutant allele burden. Adverse effects include expected toxicities such as myelosuppression and periorbital edema, but also cognitive impairment in some patients. Although considerable excitement about avapritinib exists, more data are needed to assess long-term responses and adverse effects of this novel TKI.

Project description:ObjectiveMastocytosis is a complex disorder affecting various organs. The diagnostic workup can be challenging and requires a multidisciplinary approach including the use of uncommon tests. To assess mastocytosis management around the globe, we conducted the first worldwide online survey for physicians.MethodsA 21-item questionnaire was sent out to the members of the World Allergy Organization (WAO), the Global Allergy and Asthma European Network (GA2LEN), the Urticaria (UCARE) and Angioedema (ACARE) Centers of Reference and Excellence, the German Society of Allergology and Clinical Immunology (DGAKI), and the European Mast Cell and Basophil Research Network (EMBRN) in April-June 2021.ResultsAcross 628 respondents from 79 countries 87.7% and 9.7% of physicians were allergists/clinical immunologists and/or dermatologists. Participating physicians were from all regions of the world (Europe, EU: 41.6%; North America, NA: 24.8%; Latin America, LA: 14.5%; Asia-Pacific, AP: 12.6%; and Africa/Middle East, AME: 6.5%). Only 2.2% of respondents worked at Specialized Mastocytosis Centers (SMCs) in North America or European Union. Physicians reported caring for 4 patients with mastocytosis per year, with higher numbers in European Union and Asia Pacific (5/year) compared to Latin America (2/year). Dermatologists and physicians who work at SMCs reported higher patient numbers (15/year and 80/year, respectively). Suspicion of mastocytosis in the allergology and dermatology community is commonly driven by anaphylaxis (82.9%), mastocytosis skin lesions (82.1%), or elevated tryptase levels (76.6%). Osteoporosis and gastrointestinal symptoms less often prompted suspicion of mastocytosis (21.4% and 49.9%, respectively). World Health Organisation (WHO)-diagnostic criteria and classification, regardless of the region, are only used by about 50% of physicians, with higher rates for SMCs (83.3%). Serum tryptase, bone marrow biopsy, and KIT D816V mutation analysis are included in the diagnostic workup by 90.9%, 61.5%, and 58.4% of physicians, respectively. The biggest challenges for the management of mastocytosis are the lack of more effective treatment options (51.1%), missing multidisciplinary networks (47.1%), and the lack of experience of specialists from other disciplines (39.0%).ConclusionsThe diagnostic workup for mastocytosis differs from consensus recommendations and varies between regions. This may be improved by establishing active multidisciplinary networks, increasing access to diagnostic procedures, consistently applying WHO criteria, and developing new Mastocytosis Centers of Reference and Excellence.

Project description:IntroductionMany strategies to alleviate racial/ethnic disparities in surgical care target healthcare providers. Yet limited data exists about the perception of disparities among the range of clinical staff who work in perioperative settings. Such information could help initiate conversations about disparities in perioperative care and, if necessary, implement interventions to alleviate them. Our aim was to evaluate the association between sociodemographic characteristics, clinical position (physicians and non-physicians) and perception of perioperative disparities at a large tertiary care center.MethodsWe surveyed perioperative staff at the institution using an anonymous online survey. Primary outcome was respondents' perception of disparities in perioperative care at the institution due to patients' insurance status/type, ability to speak English, education, and racial/ethnic minority status. The association between clinical position (physician vs. non-physician) and perception of disparities was assessed in bivariate and then multivariable analysis, adjusting for respondents' race, sex, age, and years at the institution. Secondary outcomes included perception of disparities in perioperative care in the United States due to patients' insurance status/type, ability to speak English, education, and racial/ethnic minority status.Results217 completed questions that could be analyzed. Among these responders, 101 were physicians (46.5%), 165 (76.0%) were white, and 144 (66.4.%) were female. Bivariate and multivariate analysis revealed that physicians had higher perception of disparities in perioperative care at the institution based on patients' ability to speak English, education, and racial/ethnic minority status. Physicians also had higher perceptions of disparities in perioperative care in the United States than non-physicians.ConclusionsPhysicians reported higher perceptions of disparities in perioperative care than non-physicians, potentially explained by differences in training or contact with patients. Such findings serve as a first step at examining and discussing disparities in perioperative care and warrant further study.

Project description:ObjectivesTo examine healthcare provider-related perceptions toward deprescribing inappropriate medications among older adults.MethodsA cross-sectional, correlational study used a convenience sample of outpatient older adults to measure their perception toward deprescribing using a Patient's Perceptions of Deprescribing (PPoD), which include 57 multiple-choice questions related to patients' sociodemographic data, health, medicines, healthcare providers, and experience of care provided by the clinic. Data were collected by a graduate nursing student from one pharmacy in a public hospital, five days per week, via in-person interviews.ResultsData were analyzed for 200 participants. The level of patient collaboration with their primary care providers (PCPs) is linked to their trust in PCPs, beliefs about medication use, PCP knowledge, and medication concerns (p < .0001). Patient involvement in medication deprescribing decision-making is also associated with trust in PCPs and willingness to stop a medication (p < .0001). Additionally, trust in PCPs is related to patient involvement in decision-making, PCP knowledge, general health, collaboration with PCPs, and receiving conflicting information about a medicine (p = .010). Lastly, PCP medication knowledge is associated with trust in PCPs, views on the importance of medicines, medication concerns, seeking help with medicines, interactions with clinical pharmacists, and being advised by a clinical pharmacist to discontinue medication (p < .0001).ConclusionsThe study found that older adults' trust in their PCP, collaboration with their PCP, involvement in the decision-making of deprescribing, and knowledge about medication are associated with clinical and medicine-related factors. Therefore, PCPs should discuss the benefits of deprescribing inappropriate medications to prevent long-term side effects. Future studies should focus on the effectiveness of evidence-based deprescribing protocols for older adults.

Dataset Information

Perceptions of patient disease burden and management approaches in systemic mastocytosis: Results of the TouchStone Healthcare Provider Survey.

Background

Methods

Results

Conclusions

Lay summary

Publications

Perceptions of patient disease burden and management approaches in systemic mastocytosis: Results of the TouchStone Healthcare Provider Survey.

Similar Datasets

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