{"id":1600,"date":"2015-03-23T14:10:14","date_gmt":"2015-03-23T14:10:14","guid":{"rendered":"https:\/\/touchneurology.com\/2015\/03\/23\/patient-reported-outcomes-an-emerging-cornerstone-of-effective-intravenous-immunoglobulin-therapy\/"},"modified":"2015-03-23T14:10:14","modified_gmt":"2015-03-23T14:10:14","slug":"patient-reported-outcomes-an-emerging-cornerstone-of-effective-intravenous-immunoglobulin-therapy","status":"publish","type":"post","link":"https:\/\/touchneurology.com\/neuromuscular-diseases\/journal-articles\/patient-reported-outcomes-an-emerging-cornerstone-of-effective-intravenous-immunoglobulin-therapy\/","title":{"rendered":"Patient-reported Outcomes\u2014An Emerging Cornerstone of Effective Intravenous Immunoglobulin Therapy"},"content":{"rendered":"

Patient-reported Outcome Measures <\/b>
The primary aim of treating disease is to give patients freedom from symptoms, disability and side effects, and the freedom to function. Objectively and reliably measuring disability and quality of life (QoL) can be challenging but using patient-reported outcome measures (PROMs), in addition to clinical examination by physician, has proved a valuable means of capturing patient status and response to treatment.1\u20133 <\/sup> <\/p>\n

In disease management, PROMS can be used to estimate symptoms (severity\/tolerability of dysfunction or symptom), function, and health-related QoL [HRQoL], such as psychologic well-being, social, and physical functioning). The US Food and Drug Administration (FDA) encourages validated PROMs as parameters that can support labeling requirements. They can be used as endpoints in clinical studies and in regular clinical use.4<\/sup> The FDA industry guidelines support the use of outcomes measures for specific neuromuscular disorders such as use of the Rasch-built Overall Disability Scale (R-ODS) in the management of chronic inflammatory demyelinating polyneuropathy (CIDP).5<\/sup> <\/p>\n

Case Example 1 <\/b>
A 70-year-old male with a 25-year history of MMN showed right ulnar neuropathy at onset and subsequently showed left foot drop, right radial, and upper trunk symptoms with episodes occurring every 6\u20138 months at which time a course of 1 g\/kg IVIG was prescribed. Following IVIG, he reported a dramatic improvement in addition to better scores on MMT. He regained ability to perform exercises including lifting weights, his hands returned to near normal function, and he reported that the greatest improvement was in his balance. Approximately 4 months after each treatment he started to weaken again and so was treated, as needed, every 6 months. The patient was asked to assess his condition at intervals before and after treatment using the form shown in Figure 1; his resultant scores are shown in Figure 2.<\/i> <\/p>\n

PROMS are also valuable for investigation of the natural history of neuromuscular disease. An example is the 15-item MG-specific QoL scale (MG-QoL15) that was developed from a larger set of 60 items and reduced to a core set.6<\/sup> In a Japanese study that included 640 consecutive patients with MG, MG-QOL15 scores correlated with prednisolone dose and diseaseseverity.7<\/sup> MG-QOL15 scores for patients receiving \u22645 mg\/day prednisolone were the same as those receiving no prednisolone whereas scores were significantly worse for those receiving >5 mg\/day prednisolone. <\/p>\n

Another example of a PROM that has provided insights into disease natural history is the Inclusion Body Myositis Functional Rating Scale (IBM-FRS), a 10-point disease-specific functional rating scale designed for use in patients with inclusion body myositis (IBM).8<\/sup> This test takes 15 minutes to perform, shows good correlation with other test measures, such as manual muscle testing (MMT), and accurately indicates disease status. The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) is another validated PROM that has been used in multiple clinical studies as well as routinely in the clinic and is used more frequency than IBM-FRS.9\u201312<\/sup> The ALSFRS consists of four domains: gross motor tasks, fine motor tasks, bulbar functions, and respiratory function, which are not equally weighted. ALSFRS scores change in a linear fashion as ALS progresses whereas that is not fully established with IBM-FRS in IBM. <\/p>\n

PROMS are also prominently included among data gathered in various patient registries, which also provide information on the natural history of neurologic disease.13,14<\/sup> PROMs form a central component of comparative effectiveness research initiatives across a range of different diseases;15\u201317<\/sup> they correlate well with assessments of HRQoL;18<\/sup> and indicate patient perceptions of illness and disease severity.19,20<\/sup> <\/p>\n

PROMs help monitor a patient over time and can be instrumental in learning about an individual\u2019s perspective in the clinic. Assessment scales such as the MG-QOL15 are designed to involve patients and include them as part of the decision-making process. PROMs are designed so patients consider their progress in a structured manner, and are useful in many chronic neuromuscular diseases with manifestations that the patient is aware of such as: MG, CIDP, multifocal motor neuropathy (MMN), myopathies, and painful neuropathy.5,21,22<\/sup> They can be used to determine the efficacy of treatment, especially where improvements can be observed quickly. PROMs, however, are less applicable to diseases in which manifestations are not so clear to the patient or where improvement is not quickly discernible.23-26<\/sup> Such diseases may include diabetes, hypertension, inherited neuropathy, and dementia. In addition, conditions that develop slowly in which the effects of disease-modifying therapy have gradual and long-term effects are also less suitable for assessment using PROMs. PROMs are also less useful where there are large gaps between assessments. It is important therefore to consider the timing of treatment and follow-up before using PROMs and before selecting which one to use. Despite these limitations, PROMs are useful for many chronic conditions.14,23,24,27<\/sup> <\/p>\n

The PROMs used in Case Example 1 were a valuable and reliable means of monitoring MMN and identifying when a dose of intravenous immunoglobulin (IVIG) is necessary or a change in treatment plan is warranted. If a patient is being treated on an as needed basis, PROMs can alert the nurse or physician when clinical intervention is necessary, and in this way assist in managing symptoms to better control the condition. <\/p>\n

A variety of useful PROMs exist for the assessment of neuromuscular disease (see Table 1<\/i>).28\u201332<\/sup> Many of these are designed for specific diseases, such as the Myasthenia Gravis Activities of Daily Living (MG-ADL)31<\/sup> scale, whereas some are applicable to a variety of diseases such as the Short Form 36-item scale (SF-36).28<\/sup> Other scales include the R-ODS (uses include CIDP and Guillain Barre Syndrome [GBS], and Neurology Quality of Life [system] [NeuroQoL] short forms). These and other patient self-assessment instruments have been assessed and validated by the National Institutes of Health PROMIS network33,34<\/sup> and by the National Institute of Neurological Disorders and Stroke Common Data Elements (CDE) and are available from their websites.35<\/sup> <\/p>\n

PROMs provide the patient perspective but are subjective\u2014the clinician must decide how much weight to give each symptom\/outcome measure versus clinical and laboratory findings. Patient circumstances (mood, environment, perception) may change over time and unrelated factors can affect PROM assessments. In addition, some patients seek attention or exaggerate symptoms, which creates artifacts and confounds results. <\/p>\n

Despite these shortcomings, PROMs work well for most patients and add value to overall assessment. It is only necessary to choose one or two of the most appropriate scales. It is important to consider what parameters are required before selecting the scale. PROMs are useful in many clinical settings and prospective studies and are likely to be increasingly used as endpoints in clinical studies in the regular use for assessment and monitoring in the clinic. <\/p>\n

Opportunities to Improve the Immunoglobulin Patient Care Continuum <\/b>
The continuum of care provided to patients receiving IVIG therapy is demonstrated in Case Example 2, which is a woman with CIDP. <\/p>\n

The choice of treatment in this case of CIDP is between IVIG and corticosteroids. The decision requires consideration or treatment efficacy, side effects\/risks, and the convenience of administration at home. Following the decision to administer IVIG at home, to maintain the patient continuum of care a Walgreens\u2019 pharmacist would perform an initial evaluation and counseling. This is designed to proactively reduce the risk for adverse events by determining a detailed medical history. This is important since IVIG therapy is associated with an increased risk for thromboembolism, renal insufficiency or failure, aseptic meningitis syndrome, anaphylaxis, and hemolytic anemia. Baseline PROM assessments of the patient revealed a continued decline in symptoms and QoL during the previous month, an ability to walk less than 10\u00a0feet, and always needing a cane or walker for mobility. <\/p>\n

The continuum of care therefore begins with the treatment plan created by the clinician. Walgreens\u2019 clinical staff can then help define the risk for adverse events and establish baseline PROM scores. If serious risks are identified, they are reported to the physician and an alternative management plan is devised. <\/p>\n

Walgreens PartnerPoint Clinical ManagementSM<\/sup>\u2014Use in Case Example <\/b>
The Walgreens IG program is a comprehensive IG management approach that utilizes a clinical management technology, Walgreens PartnerPoint Clinical Management.SM<\/sup> PartnerPoint provides patient-specific clinical data, adverse event monitoring, clinical intervention tracking, and disease-specific outcomes. PartnerPoint provides transparent, consistent communication between the home infusion clinical team and the physician. Graphical reports trending clinical outcomes based on PROMs provide the physician tools that can be used to optimize care. This can assist the physician with managing dosing, pre-medication, adjunctive therapies, and IVIG cycle management, ultimately improving patient outcomes and demonstrating efficacy of treatment. <\/p>\n

The value of Walgreens PartnerPoint Clinical ManagementSM<\/sup> can be demonstrated by the above CIDP case example. Round two of the patient\u2019s treatment consists of 1 g\/kg over 3 days every 4 weeks. One hour into the infusion the patient experiences headache, mild nausea, and flashing. The nurse halts the infusion for 30 minutes, repeats premedications, and decreases the infusion rate, but the headache persists. The pharmacist notifies the physician and for subsequent administrations the infusion rate is decreased and prehydration ordered. <\/p>\n

The details of the adverse event are entered into the Walgreens PartnerPoint Clinical ManagementSM<\/sup> system including type, severity score, management, and outcome. This information is communicated to the physician along with plots showing event incidence over time (see\u00a0Figure\u00a03<\/i>). Subsequent infusions are tolerated with no further adverse events. Over 6 months, the patient stabilizes and improves. PROMs revealed marked improvements including Inflammatory Neuropathy Cause and Treatment (INCAT) scores decreasing to 1 in the arms and legs and in R-ODs, ambulation, and impression of health status (see Figure 4<\/i>). <\/p>\n

Walgreens PartnerPoint Clinical ManagementSM<\/sup> has a number of advantages for the physician. These include improving the care continuum for the home IG patient and proactively avoiding or managing adverse events. This management system also ensures that PROMs and QoL measures are obtained monthly and that consolidated graphical reports are supplied to the prescribing physician on a regular basis or as requested. In the management of patients with neurologic disease the system provides the following advantages: <\/p>\n