Search

The American Society for Apheresis (ASFA) Journal of Clinical Apheresis (JCA) Special Issue Writing Committee is charged with reviewing, updating, and categorizing indications for the evidence-based use of therapeutic apheresis (TA) in human disease. In the Ninth Edition, the JCA Special Issue Writing Committee has incorporated systematic review and evidence-based approaches in the grading of evidence and categorization of apheresis indications to make recommendations on the use of apheresis in a wide variety of diseases and conditions. This edition has largely maintained the general layout and concept of a fact sheet introduced in the Fourth Edition (2007). Each fact sheet succinctly summarizes the evidence for the use of TA in a specific disease or medical condition. The Ninth Edition of the JCA Special Issue comprises 91 fact sheets and 166 graded and categorized indications. This includes seven new fact sheets, nine new indications on existing fact sheets, and eight changes in the category for existing indications. The Ninth Edition of the JCA Special Issue seeks to continue to serve as a key resource that guides the utilization of TA in the treatment of human disease., (© 2023 Wiley Periodicals LLC.)

Aim: This study aimed to model the financial impact of caplacizumab with therapeutic plasma exchange (TPE) + immunosuppression for patients experiencing an acute acquired thrombotic thrombocytopenic purpura (aTTP) episode versus TPE + immunosuppression, from a US hospital's perspective., Methods and Materials: We developed an economic model to estimate the impact of caplacizumab on a US hospital's budget. Cost offsets from caplacizumab utilization targeted inpatient general ward days, intensive care unit (ICU) days, and TPE utilization. Costs and event probabilities were estimated from primary data analyses of the phase 3 HERCULES trial and peer-reviewed literature or other public sources. Plan reimbursement was obtained from 2019 Medicare Fee Schedules and adjusted to represent reimbursement from different US payers. Cost of ICU and general ward utilization were estimated from Medicare Provider Analysis and Review data analyses capturing hospital discharges., Results: The model results indicate that caplacizumab leads to hospitalization cost savings of over $8,000 ($23,148 versus $14,904) along with TPE cost savings of over $14,000 ($37,150 versus $23,033) per patient. When the cost of caplacizumab and plan reimbursement are incorporated into the results, the per-patient cost of TPE + immunosuppression is $23,120 versus $70,068 for caplacizumab with TPE + immunosuppression, an incremental cost of $46,948. The model was robust to several scenario analyses; however, when limited to Medicare fee-for-service (FFS), the incremental cost of caplacizumab per patient was reduced to $4,852 due to add-on payments., Conclusions: Caplacizumab with TPE + immunosuppression is associated with an increase in costs; however, the increase is nominal among payers who provide an add-on payment consistent with that of Medicare FFS.

Background: Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare hematologic disorder that can lead to serious life-threatening medical complications., Objective: The aim of this study was to describe aTTP-related hospital resource utilization, cost, complications, and overall survival among US Medicare and non-Medicare populations following aTTP episodes prior to the US approval of caplacizumab., Methods: This retrospective study utilized administrative claims data for Medicare Fee-for-Service (FFS) beneficiaries (100% sample) and a sample of commercial, managed Medicaid [MM], Medicare Advantage [MA] plan members from the Inovalon MORE 2 Registry. aTTP patients ages 18+ were identified between 2010 and 2018 using a published validated algorithm: ≥1 hospitalization for thrombotic microangiopathy + therapeutic plasma exchange (TPE). 2,279 patients were identified; 65.2% were enrolled in Medicare FFS, 13.6% in commercial, 15.7% in MM, and 5.4% in MA. Mean hospitalization days for aTTP index episode ranged between 12 and 17 days; ∼60% of patients required intensive care. Mean payments for index hospitalization varied by payer [Medicare FFS: $29,024; MA: $12,860; commercial: $9,996 and MM: $10,470]. Among FFS patients, 15.7% died during initial hospitalization and 21.0% died within first 30 days of the event. During follow-up, 11.6-19.6% experienced aTTP-related exacerbation. Incidence rate of relapse and complications per 100 person-years was 5.6 [Medicare FFS: 3.6; MA: 8.7; commercial: 10.4 and MM: 14.7] and 16.7 [FFS: 15.5; MA: 20.5; commercial: 21.7 and MM: 19.1], respectively. Among Medicare patients with and without aTTP, mortality risk was 2.9 (95 % CI: 2.4-3.4) times higher for aTTP vs. non-aTTP patients., Conclusion: This is the first real-world study evaluating burden of illness among aTTP patients in the US across payer types. Despite being treated with TPE, patients with aTTP have lower survival rates in comparison to a matched cohort without aTTP. These findings highlight the need for more effective and novel therapies to reduce disease burden for this population.Key pointsIn US Medicare and managed care populations with aTTP between 2010 and 2018, aTTP can lead to significant utilization of ICU services due to clinical complications, and/or relapse following hospital discharge.Despite treatment with therapeutic plasma exchange, acute mortality remains high (15.7%) indicating the need for more effective and novel treatments.

Importance: Neuromyelitis optica/neuromyelitis optica spectrum disorder patients' response to therapeutic plasma exchange (TPE) is currently incompletely characterized., Objective: Our study aims to understand the clinical status improvement of neuromyelitis optica/neuromyelitis optica spectrum disorder patients treated with TPE., Design, Setting, and Participants: This is a multicenter retrospective study conducted between 1 January 2003 and 31 July 2017 at 13 US hospitals performing apheresis procedures. Subjects studied were diagnosed with neuromyelitis optica/neuromyelitis optica spectrum disorder who received TPE during presentation with acute disease., Main Outcomes and Measures: The primary outcome was clinical status improvement in patients treated with TPE. Secondary measures were procedural and patient characteristics associated with response to treatment., Results: We evaluated 114 patients from 13 institutions. There was a female predilection. The largest ethnic group affected was non-Hispanic Caucasian. The average age of diagnosis was 43.1 years. The average time to diagnosis was 3.1 years. On average, five procedures were performed during each treatment series. The most commonly performed plasma volume exchange was 1.0 to 1.25 using 5% albumin as replacement fluid. Most patients (52%) did not require an additional course of TPE and noted "mild" to "moderate" clinical status improvement. Maximal symptom improvement appeared by the fourth or fifth TPE treatment., Conclusion and Relevance: TPE improved the clinical status of patients. Adults responded more favorably than children. Procedural characteristics, including number of TPEs, plasma volume exchanged, and replacement fluid used, were similar between institutions. TPE was well-tolerated and had a low severe adverse event profile., (© 2019 Wiley Periodicals, Inc.)

The American Society for Apheresis (ASFA) regularly publishes evidence-based guidelines, with the most recent edition in 2016, to assist the requesting and/or apheresis physicians with the evaluation of therapeutic apheresis. Given that therapeutic plasma exchange (TPE) is one of the most common therapeutic apheresis procedures, in this review, we discuss the rationale of TPE in both ASFA category I (first-line therapy) and II (second-line therapy) indications. However, the ASFA Guidelines usually provide little guidance with regard to scheduling/urgency issues. Given that mobilizing resources to perform apheresis after-hours may be expensive and challenging, we classified the urgency of the procedures in this review into 3 distinct groups: emergent (i.e. TPE should be started as soon as possible, preferably within 4-6 h upon request), urgent (i.e. TPE should be initiated within 24 h of request), and routine (i.e. TPE may be performed during regular working hours) based on our experiences in clinical practices. A brief discussion of the technical aspects as well as important considerations for an apheresis consultation is also provided., (Copyright © 2019 Elsevier Ltd. All rights reserved.)

The American Society for Apheresis (ASFA) Journal of Clinical Apheresis (JCA) Special Issue Writing Committee is charged with reviewing, updating and categorizing indications for the evidence-based use of therapeutic apheresis (TA) in human disease. Since the 2007 JCA Special Issue (Fourth Edition), the committee has incorporated systematic review and evidence-based approaches in the grading and categorization of apheresis indications. This Eighth Edition of the JCA Special Issue continues to maintain this methodology and rigor in order to make recommendations on the use of apheresis in a wide variety of diseases/conditions. The JCA Eighth Edition, like its predecessor, continues to apply the category and grading system definitions in fact sheets. The general layout and concept of a fact sheet that was introduced in the Fourth Edition, has largely been maintained in this edition. Each fact sheet succinctly summarizes the evidence for the use of TA in a specific disease entity or medical condition. The Eighth Edition comprises 84 fact sheets for relevant diseases and medical conditions, with 157 graded and categorized indications and/or TA modalities. The Eighth Edition of the JCA Special Issue seeks to continue to serve as a key resource that guides the utilization of TA in the treatment of human disease., (© 2019 Wiley Periodicals, Inc.)

Background: A prior practice survey revealed variations in the management of patients with sickle cell disease (SCD) and stressed the need for comprehensive guidelines. Here we discuss: 1) common indications for red blood cell exchange (RCE), 2) options for access, 3) how to prepare the red blood cells (RBCs) to be used for RCE, 4) target hemoglobin (Hb) and/or hematocrit (Hct) and HbS level, 5) RBC depletion/RCE, and 6) some complications that may ensue., Study Design and Methods: Fifteen physicians actively practicing apheresis from 14 institutions representing different areas within the United States discussed how they manage RCE for patients with SCD., Results: Simple transfusion is recommended to treat symptomatic anemia with Hb level of less than 9 g/dL. RCE is indicated to prevent or treat complications arising from the presence of HbS. The most important goals are reduction of HbS while also preventing hyperviscosity. The usual goals are a target HbS level of not more than 30% and Hct level of less than 30%., Conclusion: Although a consensus as to protocol details may not be possible, there are areas of agreement in the management of these patients, for example, that it is optimal to avoid hyperviscosity and iron overload, that a target Hb S level in the range of 30% is generally desirable, and that RCE as an acute treatment for pain crisis in the absence of other acute or chronic conditions is ordinarily discouraged., (© 2018 AABB.)