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Patient characteristics and platelet responses at romiplostim initiation according to the duration of immune thrombocytopenia (ITP) are poorly understood. Amongst romiplostim-exposed adults with ITP lasting ≥6 months during 2009-2018 in Denmark, Sweden, and Norway, we examined characteristics at romiplostim initiation, romiplostim dosage, and durable platelet response (≥75% of measurements ≥50 × 109/L at 14-24 weeks) for subcohorts with newly diagnosed (duration <3 months), persistent (3-12 months), or chronic (>12 months) ITP initiating romiplostim. The 285 romiplostim initiators comprised 81 (28%) with newly diagnosed, 47 (16%) with persistent, and 157 (55%) with chronic ITP. More patients with newly diagnosed ITP than longer ITP duration, had low comorbidity levels, two or more prior ITP therapies, and previous bleeding requiring hospitalisation. The median romiplostim doses were similar across subcohorts. During treatment, median platelet counts were similar across subcohorts (75-76 × 109/L), and the durable platelet response was 64.6%, 52.9%, and 52.7% for newly diagnosed, persistent, and chronic ITP, respectively. After treatment cessation, the median platelet count was 138 × 109/L, 68 × 109/L, and 71 × 109/L, respectively. In conclusion, newly diagnosed patients, compared with romiplostim initiators with longer disease duration, had more severe ITP, higher frequency of durable platelet response, and higher median platelet count after cessation.
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ObjectiveTo assess use of bone-targeting agents (BTA) in patients with confirmed bone metastases (BM) from breast cancer (BC), non-small cell lung cancer (NSCLC) or prostate cancer (PC). DESIGN: Retrospective cohort study. SETTING: Regional hospital-based oncology database of approximately 2 million patients in England. PARTICIPANTS: Patients aged ≥18 years with a diagnosis of BC, NSCLC or PC as well as BM between 1 January 2007 and 31 December 2018, with follow-up to 30 June 2020 or death; BM diagnosis ascertained from recorded medical codes and unstructured data using natural language processing (NLP). MAIN OUTCOMES MEASURES: Initiation or non-initiation of BTA following BM diagnosis, time from BM diagnosis to BTA initiation, time from first to last BTA, time from last BTA to death. RESULTS: This study included 559 BC, 894 NSCLC and 1013 PC with BM; median age (Q1-Q3) was 65 (52-76), 69 (62-77) and 75 (62-77) years, respectively. NLP identified BM diagnosis from unstructured data for 92% patients with BC, 92% patients with NSCLC and 95% patients with PC. Among patients with BC, NSCLC and PC with BM, 47%, 87% and 88% did not receive a BTA, and 53%, 13% and 12% received at least one BTA, starting a median 65 (27-167), 60 (28-162) and 610 (295-980) days after BM, respectively. Median (Q1-Q3) duration of BTA treatment was 481 (188-816), 89 (49-195) and 115 (53-193) days for patients with BC, NSCLC and PC. For those with a death record, median time from last BTA to death was 54 (26-109) for BC, 38 (17-98) for NSCLC and 112 (44-218) days for PC. CONCLUSION: In this study identifying BM diagnosis from both structured and unstructured data, a high proportion of patients did not receive a BTA. Unstructured data provide new insights on the real-world use of BTA.
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Neoplasias Óseas , Neoplasias de la Mama , Carcinoma de Pulmón de Células no Pequeñas , Neoplasias Pulmonares , Neoplasias de la Próstata , Masculino , Humanos , Adolescente , Adulto , Carcinoma de Pulmón de Células no Pequeñas/tratamiento farmacológico , Carcinoma de Pulmón de Células no Pequeñas/patología , Neoplasias Pulmonares/tratamiento farmacológico , Neoplasias Pulmonares/patología , Estudios Retrospectivos , Registros Electrónicos de Salud , Neoplasias Óseas/tratamiento farmacológico , Neoplasias Óseas/secundario , Neoplasias de la Próstata/tratamiento farmacológico , Neoplasias de la Próstata/patología , Neoplasias de la Mama/tratamiento farmacológico , Neoplasias de la Mama/patología , Pulmón/patología , Reino Unido/epidemiologíaRESUMEN
PURPOSE: To describe (non)adherence with denosumab among patients with solid tumors and bone metastases. METHODS: This retrospective, observational study pooled data from two completed prospective, multicenter cohort studies (X-TREME; Study 240) in adult patients with bone metastases from primary breast, prostate, lung, kidney, or other solid cancer types and administered denosumab 120 mg in routine clinical practice in Germany and Central and Eastern Europe. The studies were conducted between May 2012 and May 2017; pooled analysis was completed in August 2021. Medication adherence was described according to a three-component consensus taxonomy: initiation (first-ever administration ≤ 90 days from bone metastasis diagnosis), implementation (actual vs prescribed dosing; optimal implementation = regular/consistent dosing), and persistence (≤ 60-day gap between administrations at 3, 6, 9, and 12 months). Descriptive analyses were conducted for each cancer type. RESULTS: The analysis included 1748 patients with solid tumors and bone metastases. Adherence with denosumab was generally high across the initiation, implementation, and persistence phases. Most patients experienced timely initiation (from 64.4% [kidney cancer] to 81.2% [breast cancer]) and optimal implementation (from 62.4% [lung cancer] to 72.5% [breast cancer]). The proportion of patients who were persistent with treatment at 6 months ranged from 41.4% (lung cancer) to 77.8% (prostate cancer). CONCLUSIONS: This study revealed variations by cancer type in the initiation, implementation, and persistence of denosumab in patients with solid tumors and bone metastases in routine clinical practice. Further cancer-specific studies are warranted to examine the determinants of (non)adherence with denosumab, and potential ways to improve medication adherence.
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Conservadores de la Densidad Ósea , Neoplasias Óseas , Neoplasias de la Mama , Neoplasias Pulmonares , Adulto , Masculino , Humanos , Denosumab/uso terapéutico , Estudios Retrospectivos , Conservadores de la Densidad Ósea/uso terapéutico , Estudios Prospectivos , Neoplasias Óseas/tratamiento farmacológico , Neoplasias Óseas/secundario , Cumplimiento de la Medicación , Neoplasias de la Mama/tratamiento farmacológico , Neoplasias de la Mama/patología , Neoplasias Pulmonares/tratamiento farmacológicoRESUMEN
INTRODUCTION: The effectiveness and safety of romiplostim were evaluated by immune thrombocytopenia (ITP) phase (newly diagnosed/persistent/chronic) at romiplostim initiation. METHODS: This is a post hoc analysis of a prospective, German, multicentre, observational study in adults with ITP who received ≥1 dose of romiplostim. Follow-up data were collected for ≤2 years. Outcomes included overall platelet response (≥1 platelet count ≥50 × 109/L at 2-24 weeks after romiplostim initiation) or durable platelet response (≥75% of measurements ≥50 × 109/L at 14-24 weeks) and adverse drug reactions (ADRs), evaluated by ITP phase. RESULTS: Data from 96 patients were analysed (newly diagnosed, n = 18; persistent, n = 25; chronic, n = 53). During the 2- to 24-week follow-up, overall platelet response was achieved in 100% (95% confidence interval: 81.5-100), 100% (86.3-100), and 96.2% (87.0-99.5) of patients with newly diagnosed, persistent, or chronic ITP, respectively, and platelet responses were durable in 88.2% (63.6-98.5), 65.0% (40.8-84.6), and 69.4% (54.6-81.7) of patients. During the 2-year follow-up, ADRs occurred in 24.0-35.8% of patients across phases. Two patients with chronic ITP experienced bone marrow ADRs; no thrombotic ADRs occurred. CONCLUSION: Romiplostim was effective and well tolerated in patients with newly diagnosed, persistent, or chronic ITP in routine clinical practice.
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Púrpura Trombocitopénica Idiopática , Receptores Fc , Trombocitopenia , Trombopoyetina , Adulto , Alemania , Humanos , Estudios Prospectivos , Púrpura Trombocitopénica Idiopática/inducido químicamente , Receptores Fc/uso terapéutico , Proteínas Recombinantes de Fusión/uso terapéutico , Trombocitopenia/tratamiento farmacológico , Trombopoyetina/uso terapéutico , Resultado del TratamientoRESUMEN
BACKGROUND: Guidelines recommend starting bone-targeted agents (BTA), such as zoledronic acid and denosumab, as soon as bone metastases (BMs) are definitively diagnosed in all patients with breast cancer (BC) or castration-resistant prostate cancer (CRPC) whether they are symptomatic or not. METHODS: Data were analyzed from 1364 patients with BC and 1161 patients with CRPC who had BMs and were receiving anti-cancer therapy in hospitals across six European countries (Belgium, France, Germany, Italy, Spain and the UK). The 731 physicians (medical oncologists or urologists) provided insights in the decision-making factors driving their management of bone health for these patients, and the patient medical records indicated how these decisions were reflected in routine clinical practice. RESULTS: Within three months of a BM diagnosis, 74% of BC and 51% of CRPC patients had initiated treatment with a BTA. Around 12% of BC and 23% of CRPC patients did not receive a BTA following BM diagnosis. Irrespective of the tumour type (BC or CRPC), most physicians prescribed either denosumab or zoledronic acid as first BTA therapy. Physicians reported bone pain as a major decision-making factor to initiate a BTA. The presence of bone complications at BM diagnosis and bone pain at BM diagnosis were found to be significant predictive factors for a BTA initiation, irrespective of tumour type. CONCLUSIONS: Despite European Society for Medical Oncology (ESMO) guidance on bone protection irrespective of symptomatic disease, not all patients with BMs received a BTA following a BM diagnosis. This suggests that clinical judgements and patients' communication of their pain to their physicians contributed to the decision to prescribe bone protection therapy in cancer patients.
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Conservadores de la Densidad Ósea , Neoplasias Óseas , Neoplasias de la Mama , Neoplasias de la Próstata , Conservadores de la Densidad Ósea/uso terapéutico , Neoplasias Óseas/tratamiento farmacológico , Neoplasias Óseas/secundario , Neoplasias de la Mama/tratamiento farmacológico , Neoplasias de la Mama/patología , Alemania , Humanos , Masculino , Neoplasias de la Próstata/patología , Ácido Zoledrónico/uso terapéuticoRESUMEN
Aim: To investigate use of the 'Managing Advanced Cancer Pain Together' conversation tool between individuals with advanced cancer and healthcare professionals (HCPs) during routine consultations. Methods: Twenty-one patients and six HCPs completed questionnaires before and after use of the tool (at their routine consultation 1 and consecutive consultation 2, respectively). Results: Patients and HCPs were satisfied with communication during both consultations. When using the tool, patients most frequently selected physical pain descriptors (95.2%), followed by emotional (81.0%), social (28.6%) and spiritual (28.6%) descriptors. Patients found the tool useful, stating that it helped them describe their pain. HCPs considered the tool difficult to incorporate into consultations. Conclusion: The study highlighted the need to consider the various aspects of cancer pain.
The Managing Advanced Cancer Pain Together conversation tool was designed to help patients with advanced cancer and their healthcare professionals (HCPs) discuss various aspects of pain (physical, emotional, social and spiritual pain) during their consultations. The tool comprises 41 words to describe pain, and patients are asked to select three words that best describe their experience. For this study, patients with advanced cancer and their HCPs completed two consultations, one without the tool and one with the tool. Overall, patients found the tool helpful and used words relating to physical (95.2%), emotional (81.0%), social (28.6%) and spiritual (28.6%) pain to describe their recent experience. HCPs reported that the tool may be difficult to use during consultation due to limited time.
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Dolor en Cáncer , Neoplasias , Dolor en Cáncer/terapia , Comunicación , Personal de Salud , Humanos , Neoplasias/complicaciones , Neoplasias/terapia , Manejo del Dolor , Relaciones Profesional-PacienteRESUMEN
BACKGROUND: The association between patient self-reported pain severity and health-related quality-of-life (HRQoL) is poorly understood. AIMS: This real-world study of symptomatic multiple myeloma (MM) patients sought to determine how pain severity from a single question asked during routine clinical consultation was associated with HRQoL. METHODS AND RESULTS: Point-in-time data on HRQoL of 330 patients with MM (median age 70 years) receiving anti-myeloma therapy in Germany and Italy from November 2017 through February 2018 were analyzed. HRQoL was assessed using validated questionnaires (Work Productivity and Activity Impairment [WPAI], European Organization for Research and Treatment of Cancer Quality of Life Questionnaire -C30 and -MY20). Physical pain severity was assessed during clinical consultation by a single question, asking patients to describe their pain as "no pain," "mild," "moderate," or "severe." Associations between patient-reported pain severity and HRQoL scores were assessed by analysis of variance or χ2 tests. Ninety-six of the 330 patients (29.1%) reported moderate to severe pain. Increase in pain severity, from "no" to "severe" pain, was associated with significantly decreased overall HRQoL (mean score 70.2 to 33.3); significant decreases in levels of physical (82.7 to 35.1), social (81.1 to 44.4), emotional (78.1 to 48.3), and role functioning (79.5 to 38.9); and increased levels of WPAI usual activity impairment (35.4 to 71.4), and fatigue burden (26.0 to 68.9) (all p < .001). CONCLUSION: Higher pain severity, based on a single self-report question, was associated with poorer HRQoL in patients with MM, thereby supporting the clinical relevance of directly asking patients to self-evaluate their pain severity.
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Dolor en Cáncer/psicología , Mieloma Múltiple/psicología , Dimensión del Dolor/métodos , Calidad de Vida , Adulto , Anciano , Estudios Transversales , Femenino , Humanos , Masculino , Persona de Mediana Edad , Índice de Severidad de la Enfermedad , Encuestas y CuestionariosRESUMEN
Aim: To describe physical, social and emotional aspects of pain self-reported by patients with multiple myeloma (MM), and patient-physician communication of physical pain. Materials & methods: We analyzed self-reported data from 330 adults receiving anti-MM therapy in Germany and Italy on health-related quality of life (European Organisation for Research and Treatment of Cancer Quality of Life Core-30 Questionnaire version 3, -MY20) and bone pain symptoms. Results: Patients experienced clinically important physical (69%), emotional (58%) and social (22%) pain. Less than three-quarters of physicians' records matched patients' perception of bone pain (71.5%), with bone pain not recorded in 19.7% of patients experiencing it. Nearly half of physicians underestimated bone pain severity. Conclusion: Patients with MM experience physical, social and emotional pain. Discordance regarding bone pain symptoms and severity was observed, suggesting the need for improved communication.
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Mieloma Múltiple , Adulto , Comunicación , Humanos , Mieloma Múltiple/complicaciones , Dolor/etiología , Calidad de Vida , Encuestas y CuestionariosRESUMEN
OBJECTIVES: Risk of infections in patients with solid cancers and bone metastases (BM) and the subsequent impact on prognosis is unclear. We examined the risk of infections among patients with cancer diagnosed with BM and the subsequent impact of infections on mortality. DESIGN: Population-based cohort study. SETTING: Danish medical databases holding information on all hospital contacts in Denmark. PARTICIPANTS: Adult patients with solid cancers and BM between 1 January 1994 and 30 November 2013. OUTCOME MEASURES: In the risk analyses, the outcome was time to hospitalisation for common severe infections, pneumonia, sepsis and urinary tract infections. In the mortality analysis, we used Cox regression to compute HRs of death, modelling infection as time-varying exposure, stratifying for primary cancer type and adjusting for age, sex and comorbidities. RESULTS: Among 23 336 patients with cancer and BM, cumulative incidences of common severe infections were 4.6%, 14.0% and 20.0% during 1 month, 1 year and 10 years follow-up. The highest incidence was observed for pneumonia, followed by urinary tract infections and sepsis. Infection was a strong predictor of 1 month mortality (adjusted HR: 2.1 (95% CI 1.8 to 2.3)) and HRs increased after 1 and 10 years: 2.4 (95% CI 2.3 to 2.6) and 2.4 (95% CI 2.4 to 2.6). Sepsis and pneumonia were the strongest predictors of death. Results were consistent across cancer types. CONCLUSION: Patients with cancer and BM were at high risk of infections, which was associated with a more than twofold increased risk of death for up to 10 years of follow-up. The findings underscore the importance of preventing infections in patients with cancer and BM.
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Neoplasias Óseas , Adulto , Neoplasias Óseas/secundario , Estudios de Cohortes , Dinamarca/epidemiología , Humanos , Incidencia , Pronóstico , Factores de RiesgoRESUMEN
OBJECTIVES: Patients with multiple myeloma (MM) experience significant delays in diagnosis due to non-specific symptomatology. The aim of this study was to characterise the frequency and timing of clinical features in the primary care setting prior to MM diagnosis. DESIGN: Population-based cohort study. SETTING: Electronic health records data of approximately 17 million patients (2006-2016) within the UK Clinical Practice Research Datalink. PARTICIPANTS: Patients aged ≥18 years with newly diagnosed MM (NDMM), no history of solid tumours and ≥2 years registration in a primary care practice prior to MM diagnosis. MAIN OUTCOME MEASURES: Clinical features and symptoms including bone pain, skeletal-related events (SREs), investigation and confirmation of MM diagnostic CRAB criteria (hyperCalcaemia, Renal impairment, Anaemia, Bone lesions) during the 2 years prior to MM diagnosis; time between symptom manifestation and/or relevant investigation and diagnosis of MM. RESULTS: Among 2646 patients with NDMM, 47.5% had a bone pain record during the 2-year period prior to MM diagnosis, mainly affecting the back. Regardless of baseline bone pain, investigations for serum calcium level were used in 36.4% of patients prior to MM diagnosis, followed by haemoglobin (65.6%) or renal function (74.1%). Median (Q1, Q3) time from first-recorded bone pain to MM diagnosis was 220 (80, 476) days. Median (Q1, Q3) time from first-recorded hypercalcaemia, renal impairment or anaemia to MM diagnosis was 23 (12, 46), 58 (17, 254) and 73 days (28, 232), respectively. An imaging investigation or referral for imaging was recorded for 60.0% of patients with bone pain/SRE and 32% without. CONCLUSIONS: Nearly half of patients diagnosed with NDMM presented with bone pain approximately 7 months prior to MM diagnosis. Investigations to evaluate all CRAB criteria, including targeted imaging, were underused. Early recognition of myeloma clinical features and optimised use of investigations in primary care may potentially expedite MM diagnosis.
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Enfermedades Óseas , Mieloma Múltiple , Insuficiencia Renal , Adolescente , Adulto , Estudios de Cohortes , Humanos , Mieloma Múltiple/diagnóstico , Mieloma Múltiple/epidemiología , Atención Primaria de SaludRESUMEN
INTRODUCTION: The incidence of cardiovascular events among cancer patients with bone metastases is poorly understood. We examined rates of cardiovascular events among cancer patients with bone metastases and mortality following such events. METHODS: Using Danish health registries, we identified all Danish cancer patients diagnosed with bone metastases (1994-2013) and followed them from bone metastasis diagnosis. We computed incidence rates (IR) per 100 person-years and cumulative incidence for first-time inpatient hospitalization or outpatient clinic visit for cardiovascular events, defined as myocardial infarction, ischemic stroke, or venous thromboembolism (VTE). We also analyzed all-cause mortality rates including cardiovascular events as time-varying exposure with adjustment for age, sex, and Charlson Comorbidity Index score. All analyses were performed overall and stratified by cancer type (prostate, breast, lung, and other). RESULTS: We included 23,113 cancer patients with bone metastases. The cumulative incidence of cardiovascular events was 1.3% at 30 days, 3.7% at 1 year, and 5.2% at 5 years of follow-up. The highest IR was observed for VTE, followed by ischemic stroke and myocardial infarction, both overall and by cancer types. Lung cancer patients with bone metastases had the highest incidence of cardiovascular events followed by prostate and breast cancer. Occurrence of any cardiovascular event was a strong predictor of death (5 years following the event, the adjusted hazard ratio was 1.8 [95% confidence interval: 1.7-1.9]). CONCLUSION: Cancer patients with bone metastases had a substantial risk of developing cardiovascular events, and these events were associated with a subsequent increased mortality. Our findings underscore the importance of continuous optimized prevention of and care for cardiovascular disease among cancer patients with bone metastases.
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Neoplasias Óseas/secundario , Accidente Cerebrovascular Isquémico/epidemiología , Infarto del Miocardio/epidemiología , Tromboembolia Venosa/epidemiología , Factores de Edad , Anciano , Neoplasias de la Mama/patología , Causas de Muerte , Estudios de Cohortes , Dinamarca/epidemiología , Femenino , Hospitalización , Humanos , Incidencia , Accidente Cerebrovascular Isquémico/mortalidad , Neoplasias Pulmonares/patología , Masculino , Persona de Mediana Edad , Infarto del Miocardio/mortalidad , Neoplasias de la Próstata/patología , Factores Sexuales , Tromboembolia Venosa/mortalidadRESUMEN
PURPOSE: To describe use of bisphosphonates in newly diagnosed multiple myeloma patients in Denmark. METHODS: Using data from the Danish National Multiple Myeloma Registry, we conducted a population-based cohort study. Among patients newly diagnosed with multiple myeloma from 2005 to 2015, we examined use of bisphosphonates at first- and at progression/second-line anti-myeloma treatment overall, by patient characteristics, and myeloma complications. RESULTS: Of 2947 patients starting first-line anti-myeloma treatment, 2207 patients (74.9%) received bisphosphonates. During a median follow-up of 27.6 (quartiles, 10.6-52.5) months, disease progression post-first-line treatment was recorded in 1546 patients, of whom 1065 (68.9%) were treated with bisphosphonates. Altogether, 80.9% of patients with and 37.6% of patients without myeloma bone disease were treated with bisphosphonates at first line and 73.0% and 42.7%, respectively, at progression/second line. Moreover, the proportion of patients treated with bisphosphonates decreased with increasing severity of renal impairment at first and at progression/second-line treatment. CONCLUSION: The proportion of patients treated with bisphosphonates as part of first- and second-line anti-myeloma treatment increased with presence of myeloma bone disease and decreased by presence and severity of renal impairment. Overall, 25% of newly diagnosed multiple myeloma patients had no record of bisphosphonate treatment, potentially indicating an unmet need.
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Conservadores de la Densidad Ósea/uso terapéutico , Neoplasias Óseas/tratamiento farmacológico , Difosfonatos/uso terapéutico , Mieloma Múltiple/tratamiento farmacológico , Anciano , Estudios de Cohortes , Dinamarca , Progresión de la Enfermedad , Femenino , Humanos , Enfermedades Renales/inducido químicamente , Masculino , Persona de Mediana Edad , Sistema de Registros , Índice de Severidad de la EnfermedadRESUMEN
PURPOSE: We evaluated the reproducibility of a study characterizing newly-diagnosed multiple myeloma (MM) patients within an electronic health records (EHR) database using different analytic tools. METHODS: We reproduced the findings of a descriptive cohort study using an iterative two-phase approach. In Phase I, a common protocol and statistical analysis plan (SAP) were implemented by independent investigators using the Aetion Evidence Platform® (AEP), a rapid-cycle analytics tool, and SAS statistical software as a gold standard for statistical analyses. Using the UK Clinical Practice Research Datalink (CPRD) dataset, the study included patients newly diagnosed with MM within primary care setting and assessed baseline demographics, conditions, drug exposure, and laboratory procedures. Phase II incorporated analysis revisions based on our initial comparison of the Phase I findings. Reproducibility of findings was evaluate by calculating the match rate and absolute difference in prevalence between the SAS and AEP study results. RESULTS: Phase I yielded slightly discrepant results, prompting amendments to SAP to add more clarity to operational decisions. After detailed specification of data and operational choices, exact concordance was achieved for the number of eligible patients (N = 2646), demographics, comorbidities (i.e., osteopenia, osteoporosis, cardiovascular disease [CVD], and hypertension), bone pain, skeletal-related events, drug exposure, and laboratory investigations in the Phase II analyses. CONCLUSIONS: In this reproducibility study, a rapid-cycle analytics tool and traditional statistical software achieved near-exact findings after detailed specification of data and operational choices. Transparency and communication of the study design, operational and analytical choices between independent investigators were critical to achieve this reproducibility.
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Registros Electrónicos de Salud , Mieloma Múltiple , Estudios de Cohortes , Humanos , Mieloma Múltiple/diagnóstico , Mieloma Múltiple/epidemiología , Reproducibilidad de los Resultados , Reino Unido/epidemiologíaRESUMEN
Hematopoietic stem cell transplantation (HSCT) is considered an effective way to prevent relapse in adults with acute lymphoblastic leukemia (ALL). This study aimed to assess general trends in the use of various types of HSCTs performed between 2001 and 2015 in Europe, based on data reported to the European Society for Blood and Marrow Transplantation registry. We also evaluated HSCT rates with respect to ALL incidence in selected countries. Altogether, 15,346 first allogeneic (n = 13,460) or autologous (n = 1886) HSCTs were performed in the study period. Comparing 2013-2015 and 2001-2003, the number of allogeneic HSCTs performed in first complete remission increased by 136%, most prominently for transplantations from unrelated (272%) and mismatched related donors (339%). The number of HSCTs from matched sibling donors increased by 42%, while the total number of autologous HSCTs decreased by 70%. Increased use of allogeneic HSCT was stronger for Philadelphia chromosome (Ph)-positive (166%) than for Ph-negative ALL (38%) and for patients aged > 55 years (599%) than for younger adults (59%). The proportion of allogeneic HSCT with reduced-intensity conditioning (RIC) increased from 6 to 27%. The age-standardized rates of allogeneic HSCT per ALL incidence varied strongly among countries. Our analysis showed a continued trend toward increased allogeneic HSCT use for adults with ALL, which may be attributed to increasing availability of unrelated donors, wider use of RIC regimens, and improving efficacy of pretransplant therapy, including tyrosine kinase inhibitors for Ph-positive ALL. Allogeneic HSCT remains a major tool in the fight against ALL in adults.
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Trasplante de Células Madre Hematopoyéticas/tendencias , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Acondicionamiento Pretrasplante/tendencias , Donante no Emparentado , Adolescente , Adulto , Factores de Edad , Aloinjertos , Autoinjertos , Trasplante de Médula Ósea , Europa (Continente) , Femenino , Humanos , Masculino , Persona de Mediana Edad , Cromosoma Filadelfia , Leucemia-Linfoma Linfoblástico de Células Precursoras/epidemiología , Leucemia-Linfoma Linfoblástico de Células Precursoras/genética , Sociedades MédicasRESUMEN
BACKGROUND: Romiplostim is a thrombopoietin-mimetic peptibody for adult refractory chronic immune thrombocytopenia (ITP). We aimed to describe ITP patients receiving romiplostim, platelet counts and romiplostim usage in UK clinical practice. METHODS: This was a retrospective cohort study of patients in the UKITP Registry who received romiplostim between October 2009 and January 2015, including data up to 6 months before romiplostim initiation through follow-up. RESULTS: Of 1440 patients in the UKITP Registry, 118 adults with primary ITP were eligible. Before romiplostim, 22% had splenectomy, 12% received platelet transfusion, 97% received ≥ 1 different ITP medication and 77% received ≥ 3. Most patients (73%) initiated romiplostim ≥ 1 year after ITP diagnosis (chronic phase). The mean duration of romiplostim treatment was 5.7 (SE 0.9) months, and the median was 1.4 months (IQR: 0.2, 6.5). Mean platelet count before romiplostim was 38 × 109 /L, rising to 103 × 109 /L within 1 month, and remaining 50-150 × 109 /L through up to 3 years of follow-up. After romiplostim, 4% of patients had splenectomy, 6% received platelet transfusion, and 57% received just one ITP medication other than romiplostim. CONCLUSION: The study provides valuable insights into the real-world use of romiplostim in primary ITP in routine practice and highlighted the timing of romiplostim initiation at different ITP disease phases.
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Púrpura Trombocitopénica Idiopática/tratamiento farmacológico , Receptores Fc/uso terapéutico , Proteínas Recombinantes de Fusión/uso terapéutico , Trombopoyetina/uso terapéutico , Adolescente , Adulto , Anciano , Anciano de 80 o más Años , Biomarcadores , Coagulación Sanguínea , Terapia Combinada , Femenino , Humanos , Masculino , Persona de Mediana Edad , Recuento de Plaquetas , Pautas de la Práctica en Medicina , Púrpura Trombocitopénica Idiopática/sangre , Púrpura Trombocitopénica Idiopática/diagnóstico , Sistema de Registros , Estudios Retrospectivos , Resultado del Tratamiento , Reino Unido , Adulto JovenRESUMEN
INTRODUCTION: Romiplostim is a subcutaneously administered thrombopoietin-receptor agonist approved in the European Union for self-administration (or administration by a caregiver) in selected adult patients with chronic primary immune thrombocytopenia refractory to other treatments. To mitigate the risk of medication errors due to self-administration, the manufacturer has implemented additional risk minimisation measures (RMM) in the form of a Home Administration Training (HAT) pack to support the training of both healthcare professionals (HCPs) (guide and checklist for patient selection and training) and patients (a preparation mat, quick guide booklet, step-by-step guide, self-administration diary and DVD/video). OBJECTIVE: The primary objective was to estimate the proportion of patients/caregivers who administered romiplostim correctly after HAT pack training. METHODS: A multicentre observational study was conducted to evaluate the effectiveness of the HAT pack by recording data on a standardised collection form during direct observation of patients/caregivers in the act of administering romiplostim at the first standard-of-care visit 4 weeks after training with the HAT pack. RESULTS: Among the 40 patients/caregivers enrolled across 12 study centres in eight European countries, 35 [87.5%; 95% confidence interval (CI) 73.9-94.5] administered romiplostim correctly, and five (12.5%; 95% CI 5.5-26.1) did not. CONCLUSION: The correct administration of romiplostim by most patients/caregivers supports the effectiveness of the HAT pack as an additional risk minimisation tool in the population and setting of this study.