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WO2024227120A1 - Systèmes et procédés de surveillance de caractéristiques de qualité de vie (qol) chez des patients pnh traités avec des inhibiteurs de la voie du complément - Google Patents

Systèmes et procédés de surveillance de caractéristiques de qualité de vie (qol) chez des patients pnh traités avec des inhibiteurs de la voie du complément Download PDF

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WO2024227120A1
WO2024227120A1 PCT/US2024/026719 US2024026719W WO2024227120A1 WO 2024227120 A1 WO2024227120 A1 WO 2024227120A1 US 2024026719 W US2024026719 W US 2024026719W WO 2024227120 A1 WO2024227120 A1 WO 2024227120A1
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patient
patients
qol
pnh
epros
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Ioannis C. Tomazos
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Alexion Pharmaceuticals Inc
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Alexion Pharmaceuticals Inc
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    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16H—HEALTHCARE INFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR THE HANDLING OR PROCESSING OF MEDICAL OR HEALTHCARE DATA
    • G16H10/00—ICT specially adapted for the handling or processing of patient-related medical or healthcare data
    • G16H10/20—ICT specially adapted for the handling or processing of patient-related medical or healthcare data for electronic clinical trials or questionnaires
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16H—HEALTHCARE INFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR THE HANDLING OR PROCESSING OF MEDICAL OR HEALTHCARE DATA
    • G16H20/00—ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance
    • G16H20/10—ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16H—HEALTHCARE INFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR THE HANDLING OR PROCESSING OF MEDICAL OR HEALTHCARE DATA
    • G16H20/00—ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance
    • G16H20/70—ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to mental therapies, e.g. psychological therapy or autogenous training
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16H—HEALTHCARE INFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR THE HANDLING OR PROCESSING OF MEDICAL OR HEALTHCARE DATA
    • G16H50/00—ICT specially adapted for medical diagnosis, medical simulation or medical data mining; ICT specially adapted for detecting, monitoring or modelling epidemics or pandemics
    • G16H50/30—ICT specially adapted for medical diagnosis, medical simulation or medical data mining; ICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indices; for individual health risk assessment
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16H—HEALTHCARE INFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR THE HANDLING OR PROCESSING OF MEDICAL OR HEALTHCARE DATA
    • G16H50/00—ICT specially adapted for medical diagnosis, medical simulation or medical data mining; ICT specially adapted for detecting, monitoring or modelling epidemics or pandemics
    • G16H50/20—ICT specially adapted for medical diagnosis, medical simulation or medical data mining; ICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems

Definitions

  • Paroxysmal nocturnal hemoglobinuria is a rare blood disease characterized by the destruction of red blood cells in the blood vessels and activation of white blood cells and platelets. This can cause poor quality' of life, organ damage and potentially life- threatening blood clots.
  • Ravulizumab and eculizumab are treatments for patients with PNH known as ‘complement inhibitors’ which work by binding to a protein in the immune system (also known as ‘complement component 5’). Although clinical studies have subjectively shown that these treatments improve the quality 7 of life of patients w ith PNH. the impact on their tiredness, sleep, physical and mental health, and ability 7 to work has not been fully understood.
  • FIG. 1 illustrates a block diagram of a system for determining the efficacy of treatment for paroxysmal nocturnal hemoglobinuria (PNH) in improving quality of life (QoL), according to some aspects.
  • PNH paroxysmal nocturnal hemoglobinuria
  • FIG. 2 illustrates a method for determining the efficacy of treatment for paroxysmal nocturnal hemoglobinuria (PNH) in improving quality of life (QoL), according to some aspects.
  • FIG. 3 illustrates baseline demographics and clinical characteristics of patients enrolled in a study performed in accordance with aspects of the present disclosure. “IQR” refers to interquartile range; “PNH” refers to paroxysmal nocturnal hemoglobinuria; “SD” refers to standard deviation.
  • FIG. 4 illustrates a summary of patient recorded outcome (PRO) measures in patients with PNH treated with ravulizumab compared with the US general population, according to a study performed in accordance with aspects of the present disclosure. Values were collected longitudinally for each participant, and estimated marginal means across multiple observations per participant are shown. Error bars represent standard deviation.
  • TACIT refers to Functional Assessment of Chronic Illness Therapy
  • PRO refers to patient-reported outcome
  • PROMIS refers to Patient-Reported Outcomes Measurement Information System
  • WPAI-SHP refers to Work Productivity and Activity Impairment Questionnaire - Specific Health Problem.
  • the population normative values are 43.7 (females) and 44.7 (males) for FACIT - Fatigue, 48.5 for PROMIS Global Physical Health Short Form, 50.0 for PROMIS sleep measures and 17.0 for WPAI-SHP.
  • FIG. 5 illustrates various continuous monitoring metrics in patients with PNH treated with ravulizumab compared with the same metncs from the US general population, according to a study performed in accordance with aspects of the present disclosure. Dashed lines represent US general population normative values, “bpm” refers to beats per minute; “PNH” refers to paroxysmal nocturnal hemoglobinuria; “SD” refers to standard deviation.
  • FIG. 5A shows monitored heart rates, wherein general population normative values for resting heart rate range from 50 bpm to 80 bpm with the mean value at 65.5 bpm.
  • FIG. 5B shows monitored daily step counts, wherein general population normative values for daily steps range from 5003 steps/day to 18,425 steps/day with the mean value of 7271 steps/day.
  • FIG. 5C shows measurement of sleep duration, wherein general population normative value for mean (SD) sleep duration is 7.2 (1.0) hours.
  • FIG. 6 illustrates a summary of passively collected data in a study performed in accordance with aspects of the present disclosure, “bpm” refers to beats per minute; “SD” refers to standard deviation.
  • FIG. 7 illustrates a block diagram of example components of a computer system, according to aspects of the present disclosure.
  • Paroxysmal nocturnal hemoglobinuria is a rare and chronic blood disorder characterized by destruction of red blood cells, blood clots, and impaired bone marrow functions. These symptoms have a significant impact on patient quality’ of life (QoL) as it typically presents in early adulthood and continues throughout the patient’s life.
  • the complement component 5 (C5) inhibitors ravulizumab and eculizumab have been approved for treating patients with PNH. These treatments produce effects including, but not limited to, the reduction or cessation in fatigue, abdominal pain, dyspnea, dysphagia, chest pain, erectile dysfunction, and major adverse vascular events (MAVEs).
  • PROs Patient- reported outcomes (PROs) capturing QoL or fatigue in patients receiving treatment for PNH have been described in clinical trials. However, data on sleep patterns, physical and mental health, daily activity and work productivity are currently limited. Accordingly, there is a paucity of data regarding the relationship between PROs and activities of daily living.
  • PROs give insight into a patient’s subjective QoL, objective QoL metrics and data are lacking. Yet it is important for researchers to also understand the quantifiable physiological effects of PNH treatments on a patient to determine QoL.
  • methods for collecting data to determine QoL included surveys or questionnaires sent to the patient periodically throughout the clinical trial, which requires active participation by the patient and intervention on the part of the researchers.
  • surveys collected periodically throughout the clinical trial do not provide an accurate representation of a patient’s QoL because they do not account for potential biases of patients and reporting may be inconsistent between patients since it is subjective from patient to patient.
  • the treatments for PNH are periodic infusions rather than a daily medication, meaning periodically administering a survey to patients does not factor in changes in QoL that occur throughout the period of treatment.
  • a system has been developed that can objectively determine a patient’s QoL using PROs in conjunction with continuous, non-interventional physiological data, which together provide both subjective and objective health data to give a comprehensive view of a patient’s QoL while being treated for PNH. Not only is this important in clinical trial analysis, but in other aspects it may be used to provide healthcare providers with vital and timely information regarding their patient’s QoL throughout PNH treatment. The system may further be used by healthcare providers as an interventional mechanism to detect when QoL is significantly decreasing in a patient being treated for PNH.
  • This system provides specific insight into the effects of a PNH treatment as symptoms of PNH directly impact QoL. allowing for the determination of whether a selected PNH treatment for a given patient is sufficient such that the patient responds positively or whether the treatment should be joined with or replaced by a different therapy.
  • the PNH treatments may produce a shift towards normal levels of a hemolysis-related hematologic biomarker selected from the group consisting of free hemoglobin, haptoglobin, reticulocyte count, PNH red blood cell (RBC) clone, and D- dimer.
  • the treatment may produce a shift towards normal levels of a chronic disease biomarker selected from the group comprising estimated glomerular filtration rate (eGFR) and spot urine: albumin; and creatinine and plasma brain natriuretic peptide (BNP).
  • a chronic disease biomarker selected from the group comprising estimated glomerular filtration rate (eGFR) and spot urine: albumin; and creatinine and plasma brain natriuretic peptide (BNP).
  • the treatment may result in terminal complement inhibition such that the need for blood transfusions is reduced.
  • the treatment may also result in a reduction or elimination of breakthrough hemolysis during treatment and reduce lactate dehydrogenase (LDH) levels to normal or near normal levels.
  • LDH lactate dehydrogenase
  • some PNH treatments have been shown to increase a patient’s QoL score by at least 7 points.
  • determining efficacy of a PNH treatment on a patient’s QoL provides researchers and healthcare providers with valuable insight into the efficacy of the PNH treatment, as the treatment as described treats symptoms that negatively impact a patient’s QoL. Further, if the system indicates that a patient’s QoL is not sufficiently improved by the chosen treatment (e.g., anti-C5 treatment), the chosen treatment may be joined with or replaced by another treatment (e.g., a Factor D inhibitor (e.g., danicopan) or a C3 inhibitor (e.g., pegcetacoplan). The efficacy of the replacement or co-therapy can then be determined using the patient's QoL using the methods described herein.
  • a Factor D inhibitor e.g., danicopan
  • C3 inhibitor e.g., pegcetacoplan
  • subjectively-reported data may be combined with continuously monitored passive data to determine the QoL of a given patient undergoing treatment for a complement-mediated disease. Analyzing both subjectively-reported data and passive data limits potential bias that may be associated with the subjectively- reported data.
  • a small group of patients who were receiving either ravulizumab or eculizumab for the treatment of PNH was followed over the course of 32 weeks. The aim was to understand patient experiences of their tiredness, sleep, physical and mental health, and ability to work. Patients completed forms at regular time points about their experiences, and data (including steps, heart rate and sleep patterns) were recorded in real time on a FitbitTM wearable device. The results of the forms and the Fitbit TM data were then compared with the values for the US general population.
  • FIG. 1 illustrates an example system for determining the efficacy of PNH treatments in improving quality of life QoL using PROs and continuous, non- interventional physiological data, although other systems with similar functionality may alternatively be used.
  • System 100 includes a QoL server 102, which is a server configured to receive patient health data, transmit PROs surveys, and analyze patient health data. In some aspects, patient health data is physiological data and PROs survey responses.
  • QoL server 102 may be communicatively coupled to patient device 106 and wearable device server 1 10 via one or more networks 124. In other aspects, the QoL server may be communicatively coupled to wearable device 114 via network 124.
  • QoL server 102 includes a processor 120 and memory 122.
  • Processor 120 may be a processing device, such as computer system 700 described with reference to FIG. 7 below.
  • Processor 120 may include QoL analyzer module 104 which is configured to perform analysis on patient health data to determine QoL of a patient, as described in further detail below;
  • Patient QoL database 116 is communicatively coupled (e.g., via wired or wireless direct or indirect connection) to QoL server 102.
  • Data in patient QoL database 116 includes, but is not limited to. a patient's health data, demographics, clinical characteristics, and QoL score.
  • Data in patient QoL database 116 may be provided to QoL analyzer module 104 to identify a patient’s QoL score or determine a QoL score for a plurality of patients being treated for PNH.
  • Global health database 118 is communicatively coupled (e.g., via wired or wireless direct or indirect connection) to QoL server 102.
  • Data in global health database 118 includes, but is not limited to, global health averages for QoL in healthy individuals.
  • Data in global health database 118 may be provided to QoL analyzer module 104 to compare global QoL scores to patients’ QoL scores from patient QoL database 116 in order to determine whether patients being treated for PNH are within the global range for QoL.
  • Network 124 may be of any suitable type, including individual connections via the internet such as cellular or WiFi networks.
  • network 124 may connect terminals, services, and mobile devices using direct connections such as radiofrequency identification (RFID), near-field communication (NFC), BluetoothTM, low- energy BluetoothTM (BLE), WiFiTM, ZigBeeTM, ambient backscatter communications (ABC) protocols, USB, or LAN.
  • RFID radiofrequency identification
  • NFC near-field communication
  • BLE low- energy BluetoothTM
  • WiFiTM WiFiTM
  • ZigBeeTM ZigBeeTM
  • ABS ambient backscatter communications
  • USB or LAN
  • Network 124 may include any type of computer networking arrangement used to exchange data.
  • network 124 may be the internet, a private data network, virtual private network using a public network, and/or other suitable connection(s) that enables components in system 100 to send and receive information between the components of system 100.
  • Network 124 may also include a public switched telephone network ('‘PSTN”) and/or a wireless network.
  • PSTN public switched telephone network
  • Patient device 106 is communicatively coupled to QoL server 102 via network 124.
  • Patient device 106 can be any type of computing device, including a laptop, a desktop, a smartphone, a tablet computer, or a wearable computer (such as a smartwatch or an augmented reality or virtual reality headset).
  • patient device 106 also includes a processor and persistent, non-transitory and volatile memory.
  • the processors can include one or more central processing units, graphic processing units, or any combination thereof.
  • Patient device 106 may be a processing device, such as computer svstem 700 described with reference to FIG. 7 below.
  • Patient device 106 includes a survey application 108.
  • Survey application 108 may be a web application hosted by QoL server 102.
  • Survey application 108 provides a PROs survey, specifically electronic PROs (ePROs), to a patient.
  • the ePROs survey may include a plurality of surveys that assess physical health, mental health, and sleep respectively.
  • the ePROs survey may include one survey that assesses physical health, mental health, and sleep in a single survey.
  • subjectively-reported data may be reported on a routine basis, such as daily, weekly, biweekly, or other regular, periodic basis.
  • different subjectively- reported data may be reported on a different schedule or at different times.
  • subjectively-reported data may include one or more of functional assessment of chronic illness therapy (FACIT) fatigue data, patient-reported outcomes measurement information system (PROMIS) data (including one or more of PROMIS - sleep-related impairment, PROMIS - sleep disturbance, PROMIS - global physical health, and PROMIS - global mental health), and work productivity and activity impairment questionnaire - specific health problem (WPAI-SHP).
  • FACIT chronic illness therapy
  • PROMIS patient-reported outcomes measurement information system
  • WPAI-SHP work productivity and activity impairment questionnaire - specific health problem
  • FACIT includes a questionnaire (e.g., a 13-item questionnaire) developed to assess fatigue and validated in patients with chronic illnesses. In some aspects, it is scored on a scale of 0-52, with lower scores indicating worse fatigue.
  • a questionnaire e.g., a 13-item questionnaire
  • PROMIS Sleep-Related Impairment is a questionnaire (e.g., a 4- item questionnaire) that assesses sleep uality over a given time period (e.g., the past 7 days).
  • Raw survey scores may be calibrated to generate a standardized score, e.g. from 0- 100. Higher scores relative to the general population (mean score of 50) may represent worse sleep-related impairment.
  • PROMIS Sleep Disturbance is a questionnaire (e.g., a 4-item questionnaire) that assesses sleep quality over a given time period (e.g., the past 7 days).
  • Raw survey scores may be calibrated to generate a standardized score, e.g.. from 0-100. Higher scores relative to the general population (mean score of 50) may represent worse sleep disturbances.
  • PROMIS Global Physical Health is a questionnaire (e.g., a 2- item questionnaire) that assesses participants’ physical health over a given time period (e.g., the previous 7 days), and includes shorter, psychometrically validated variants of the PROMIS Physical Health scale.
  • raw survey scores may be calibrated against the US general population (which has a mean [standard deviation, SD] score of 50) to generate standardized score ranging, e.g., from 0 to 100.
  • SD standard deviation
  • higher scores relative to the general population may represent better physical health.
  • PROMIS Global Mental Health is a questionnaire (e.g., a 2-item questionnaire) that assesses participants mental health over a given time period (e.g., the previous 7 days), and includes shorter, psychometrically validated variants of the PROMIS Mental Health scale.
  • raw survey scores may be calibrated against the US general population (which has a mean [standard deviation, SD] score of 50) to generate standardized score ranging, e.g., from 0 to 100.
  • SD standard deviation
  • higher scores relative to the general population may represent better mental health.
  • WPAI-SHP is a questionnaire (e.g., a 6-item questionnaire) that yields four types of scores based on absenteeism (work time missed), presenteeism (impairment at work/reduced effectiveness at work), work productivity loss, and activity impairment.
  • WPAI-SPH outcomes may be expressed as impairment percentages (0- 100%), with higher numbers indicating greater impairment and less productivity 7 .
  • Wearable device 114 may be any device that may be communicatively coupled to a wearable device API 112, which is communicatively coupled (e.g., via wired or wireless direct or indirect connection) to wearable device server 108.
  • Wearable device API 112 enables wearable device server 108 to receive physiological data collected by wearable device 114.
  • Wearable device 114 may be used to monitor physiological features that correlate with QoL in patients with PNH.
  • patient experiences during treatment with C5 inhibitors for PNH may be characterized using real-world data corresponding to those physiological features, collected from wearable device 114. This real-world data may be optionally combined with data from ePROs.
  • wearable device 114 may be a digital wearable device, for example and without limitation, a FitBitTM device, an Apple WatchTM, or the like, which passively collects real-world data.
  • the real-world data may include, for example and without limitation, one or more of resting heart rate, daily number of steps, or sleep duration.
  • continuously (e.g., at a consistent and routine frequency) monitored passive data from a significant portion of each day, including one or more of resting heart rate, daily number of steps, or sleep duration may be used to determine a QoL of a given patient undergoing treatment for a complement- mediated disease.
  • the significant portion of each day may be, for example, 10 hours/day.
  • FIG. 2 illustrates a method 200 for determining the efficacy of PNH treatment in improving QoL using both subjective and passively-collected objective data.
  • the method may be used in clinical trials or by a healthcare provider in order to determine whether the QoL of a patient receiving PNH treatment is within the global QoL range.
  • Method 200 will be described with reference to system 100 illustrated in FIG. 1, although other systems with similar functionality may alternatively be used. While the present disclosure uses PNH as an example throughout, in some aspects method 200 may similarly be used to determine the QoL of patients receiving treatment for a different complement-mediated disease.
  • Method 200 may be performed by QoL server 102, as illustrated in FIG. 1.
  • a cohort of patients is determined based on demographics and clinical characteristics of a plurality of patients receiving treatment for PNH.
  • FIG. 3 illustrates an example of baseline demographics and clinical characteristics of an example cohort of patients.
  • the cohort of patients may be determined by QoL serv er 102 using patient data from patient QoL database 116.
  • QoL server 102 transmits ePROs surveys to each patient in the cohort of patients at predetermined time intervals. These surveys may include, but are not limited to, FACIT - fatigue data, PROMTS - sleep-related impairment, PROMIS - sleep disturbance, PROMIS - global physical health, and PROMIS - global mental health, and WPAI-SHP.
  • the ePROs surveys may be transmitted on a routine basis, such as daily, weekly, biweekly, or other regular, periodic basis to each patient. In some aspects, different ePROs surveys may be transmitted on a different schedule or at different times.
  • QoL server 102 receives each patient’s ePROs data corresponding to each patient’s response to the ePROs survey.
  • the ePROs data can be received from patient device 106 promptly after the patient completes the survey.
  • the ePROs data can be stored temporarily or permanently on patient device 106 and received after all of the ePROs surveys transmitted at the predetermined time intervals have been completed by the patient and/or are otherwise ready for transmission.
  • QoL server 102 While QoL server 102 is transmitting and receiving ePROs data at predetermined time intervals, at step 208, QoL server 102 is simultaneously identifying passive physiological data continuously collected during a period of time by a wearable device worn by the patient.
  • the wearable device is wearable device 114.
  • QoL server 102 uses the data collected by the wearable device, QoL server 102 calculates heart rate, identifies a daily number of steps, and calculates sleep duration for each patient in the cohort of patients. This non-interventional, continuous identification of data allows researchers or healthcare providers to understand the physiological effects of PNH treatment, while also removing the need for frequent intervention from the researchers or healthcare providers to obtain that data.
  • FIGs. 5A-C provide examples of the comprehensive view provided by continuous data collection by the wearable device.
  • QoL server 102 analyzes ePROs data and the passive physiological data for each patient in the cohort of patients and determines a QoL score for the cohort of patients based on the analysis.
  • the QoL score for the cohort of patients is compared to global QoL scores of healthy individuals. This comparison allows researchers to further understand the overall effect PNH treatment has on a patient’s QoL and determine whether treatment for PNH is effective in increasing QoL in a patient or cohort of patients.
  • C5 inhibitors used as therapy for PNH, and more specifically to ravulizumab
  • a skilled artisan will recognize that the provided disclosure for quality of life monitoring is equally applicable to other C5 inhibitors, such as eculizumab, eculizumab biosimilars, Wheatlimab, crovalimab, tesidulomab, CAN106, KP-104, and cemdisiran.
  • complement factor D e.g., danicopan, vermicopan
  • complement factor B e.g., iptacopan (LNP023), NM-8074
  • MBL associated serine protease MASP1
  • complement factor P CMP
  • complement C3 e.g., pegcetacoplan (APL-2), AMY-101 (compstatin 40), ARO-C3
  • the conducted study was a 32-week prospective, non-interventional, observational cohort study in adult patients (> 18 years) with PNH receiving C5 inhibitors in the United States. Data collected regarding baseline demographics and clinical characteristics were self-reported by the participants. In total, 28 adult patients were enrolled in the study (see Table 1). Owing to the limited number of eculizumab-treated patients, analyses were only performed for ravulizumab-treated patients.
  • IQR interquartile range
  • PNH paroxysmal nocturnal hemoglobinuria
  • SD standard deviation within Table 1.
  • Body mass index kg/m 2 , median 26.2 (23.3-28.8) 26.2 (25.1-30.5) 26.2 (23.4-29.0)
  • Lactate dehydrogenase level U/L, 226 (187-266) 233 (219-330) 230 (192-270) median (IQR)
  • ravulizumab-treated patients had a median lactate dehydrogenase level of 226 U/L and a median hemoglobin level of 11.15 g/dl. In the 3 months prior to baseline, 96% of patients had no blood transfusion.
  • Continuous activity data was passively collected via FitbitTM and included physical activity, sleep pattern, and heart rate data.
  • the minimum number of consecutive hours of device wearing required to qualify as passive data collection was 10 hours/day.
  • ePRO data were collected weekly by survey, and included:
  • PROMIS Patient-Reported Outcomes Measurement Information System
  • PROMTS Sleep-Related Impairment
  • PROMIS Sleep Disturbance
  • PROMIS Global Physical Health
  • PROMIS Global Mental Health
  • FIG. 4 provides a summary 7 of PRO measures in patients with PNH treated with ravulizumab compared with the US general population. PRO scores were within US general population normative values.
  • Each infusion occurs on a particular time cycle, and so the values prior to infusion, at infusion, and after infusion can be compared to determine improvement, decline, or stability.
  • SD mean
  • step count was comparable on the 2 days before or after infusion.
  • sleep duration was also a slight decrease in sleep duration on the day of infusion to 7.5 (1.6) hours, but this increased to 8.3 (1.9) hours on the day after infusion.
  • FIG. 6 illustrates passively collected data in ravulizumab-treated patients in the conducted study.
  • a decrease to 6313 (4183) steps was observed on the day of infusion.
  • FIG. 7 shows a computer system 700, according to some aspects.
  • Various aspects and components therein, such as system 100 and/or method 200, can be implemented, for example, using computer system 700 or any other well-known computer systems, such that the computer system, when programmed according to aspects described herein, becomes a special purpose machine.
  • computer system 700 can comprise one or more processors (also called central processing units, or CPUs), such as a processor 704.
  • processors also called central processing units, or CPUs
  • Processor 704 can be connected to a communication infrastructure or bus 706.
  • one or more processors 704 can each be a graphics processing unit (GPU).
  • a GPU is a processor that is a specialized electronic circuit designed to process mathematically intensive applications.
  • the GPU can have a parallel structure that is efficient for parallel processing of large blocks of data, such as mathematically intensive data common to computer graphics applications, images, videos, etc.
  • computer system 700 can further comprise user input/output device(s) 703, such as monitors, keyboards, pointing devices, etc., that communicate with communication infrastructure 706 through user input/output interface(s) 702.
  • Computer system 700 can further comprise a main or primary memory 708, such as random access memory (RAM).
  • Main memory 7 708 can comprise one or more levels of cache.
  • Main memory 7 708 has stored therein control logic (i. e. , computer software) and/or data.
  • computer system 700 can further comprise one or more secondary storage devices or memory 710.
  • Secondary memory 710 can comprise, for example, a hard disk drive 712 and/or a removable storage device or drive 714.
  • Removable storage drive 714 can be a floppy disk drive, a magnetic tape drive, a compact disk drive, an optical storage device, tape backup device, and/or any other storage device/drive.
  • Removable storage drive 714 can interact with a removable storage unit 718.
  • Removable storage unit 718 can comprise a computer usable or readable storage device having stored thereon computer software (control logic) and/or data.
  • Removable storage unit 718 can be a floppy disk, magnetic tape, compact disk, DVD, optical storage disk, and/ any other computer data storage device.
  • Removable storage drive 714 reads from and/or writes to removable storage unit 718 in a well-known manner.
  • secondary’ memory’ 710 can comprise other means, instrumentalities or other approaches for allowing computer programs and/or other instructions and/or data to be accessed by computer system 700.
  • Such means, instrumentalities or other approaches can comprise, for example, a removable storage unit 722 and an interface 720.
  • the removable storage unit 722 and the interface 720 can comprise a program cartridge and cartridge interface (such as that found in video game devices), a removable memory’ chip (such as an EPROM or PROM) and associated socket, a memory stick and USB port, a memory card and associated memory card slot, and/or any other removable storage unit and associated interface.
  • computer system 700 can further comprise a communication or network interface 724.
  • Communication interface 724 enables computer system 700 to communicate and interact with any combination of remote devices, remote networks, remote entities, etc. (individually and collectively referenced by reference number 728).
  • communication interface 724 can allow computer system 700 to communicate with remote devices 728 over communications path 726, which can be wired and/or wireless, and which can comprise any combination of LANs, WANs, the Internet, etc.
  • Control logic and/or data can be transmitted to and from computer system 700 via communications path 726.
  • a non-transitory, tangible apparatus or article of manufacture comprising a non-transitor ', tangible computer useable or readable medium having control logic (software) stored thereon is also referred to herein as a computer program product or program storage device.
  • control logic software stored thereon
  • control logic when executed by one or more data processing devices (such as computer system 700). causes such data processing devices to operate as described herein.

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Abstract

L'invention concerne des procédés, des systèmes et des supports lisibles par ordinateur pour déterminer l'efficacité d'un traitement d'hémoglobinurie paroxystique nocturne (PNH) dans l'amélioration de la qualité de vie (QoL). Selon certains aspects, le système utilise des résultats rapportés de patient collectés électroniquement (ePRO) et des données de santé passives provenant d'un dispositif à porter sur soi afin de déterminer la QoL d'un patient traité pour une PNH par rapport à des moyennes de QoL globales.
PCT/US2024/026719 2023-04-27 2024-04-28 Systèmes et procédés de surveillance de caractéristiques de qualité de vie (qol) chez des patients pnh traités avec des inhibiteurs de la voie du complément Pending WO2024227120A1 (fr)

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US202363498787P 2023-04-27 2023-04-27
US63/498,787 2023-04-27
US202463572781P 2024-04-01 2024-04-01
US63/572,781 2024-04-01

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Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010036700A1 (fr) * 2008-09-24 2010-04-01 Biancamed Ltd. Surveillance sans contact et à contact minimal des paramètres de qualité de vie pour évaluation et intervention
US20200254092A1 (en) * 2017-10-26 2020-08-13 Alexion Pharmaceuticals, Inc. Dosage and administration of anti-c5 antibodies for treatment of paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus)
WO2022011086A1 (fr) * 2020-07-09 2022-01-13 Alexion Pharmaceuticals, Inc. Dosage et administration d'anticorps anti-c5 pour le traitement de l'hémoglobinurie paroxystique nocturne (pnh) chez des patients pédiatriques

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010036700A1 (fr) * 2008-09-24 2010-04-01 Biancamed Ltd. Surveillance sans contact et à contact minimal des paramètres de qualité de vie pour évaluation et intervention
US20200254092A1 (en) * 2017-10-26 2020-08-13 Alexion Pharmaceuticals, Inc. Dosage and administration of anti-c5 antibodies for treatment of paroxysmal nocturnal hemoglobinuria (pnh) and atypical hemolytic uremic syndrome (ahus)
WO2022011086A1 (fr) * 2020-07-09 2022-01-13 Alexion Pharmaceuticals, Inc. Dosage et administration d'anticorps anti-c5 pour le traitement de l'hémoglobinurie paroxystique nocturne (pnh) chez des patients pédiatriques

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
WIESTLER MIRIAM ET AL: "Quality of Life Is Associated With Wearable-Based Physical Activity in Patients With Inflammatory Bowel Disease: A Prospective, Observational Study", CLINICAL AND TRANSLATIONAL GASTROENTEROLOGY, vol. 10, no. 11, 1 November 2019 (2019-11-01), pages e00094, XP093187730, ISSN: 2155-384X, Retrieved from the Internet <URL:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6890277/pdf/ct9-10-e00094.pdf> DOI: 10.14309/ctg.0000000000000094 *

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