This survey study investigates clinical care delivery practices and challenges in blinatumomab treatment among pediatric patients with B-cell acute lymphoblastic leukemia.
Key Points
Question
What are the clinical care delivery practices and challenges for blinatumomab in the treatment of childhood B-cell acute lymphoblastic leukemia?
Findings
In this survey study among 147 US member institutions of the Children’s Oncology Group in 44 states, most were using blinatumomab as standard therapy for pediatric B-cell acute lymphoblastic leukemia, but many centers had no home care company options.
Meaning
These findings suggest that home care delivery of pediatric blinatumomab represents a health care infrastructure issue that has implications for family burden, hospital resource burden, and the capacity to scale pediatric outpatient care delivery beyond blinatumomab.
Abstract
Importance
Blinatumomab, a novel immunotherapy administered as a 28-day continuous infusion, has fundamentally shifted the treatment paradigm for pediatric B-cell acute lymphoblastic leukemia (B-ALL) and is now considered a component of standard therapy for the most common childhood cancer. However, the care delivery challenges in transitioning from an experimental agent on a clinical trial to widespread clinical implementation are unknown.
Objective
To characterize the clinical landscape of pediatric blinatumomab care-delivery practice and challenges in the US from the perspective of treating centers.
Design, Setting, and Participants
This survey study was conducted from February to March 2025 among US member institutions of the Children’s Oncology Group (COG) across 44 states.
Exposure
Institutional characteristics, including participation in the National Cancer Institute Community Oncology Research Program (NCORP), US census region, site-reported annual pediatric ALL patient volume, and prior blinatumomab experience, were assessed.
Main Outcomes and Measures
Incorporation of blinatumomab as standard therapy by pediatric B-ALL subtype; major outpatient care-delivery challenges defined as 4 or 5 on a 5-point Likert scale by more than 25% of centers.
Results
Of 195 active US COG member institutions, 147 centers completed the survey and were successfully matched with a unique COG identifier, among which 35 institutions (23.8%) were NCORP participants. There were 32 institutions (21.8%) in Midwest, 30 institutions (20.4%) in Northeast, 60 institutions (40.8%) in South, and 25 institutions (17.0%) in West census regions. Most centers reported using blinatumomab as their institutional standard therapy for National Cancer Institute standard risk–average (134 centers [91.2%]), standard risk–high (144 centers [98.0%]), and high-risk (140 centers [95.2%]) B-ALL. Fewer centers reported using blinatumomab for infant (83 centers [56.5%]) or Philadelphia chromosome–positive (96 centers [65.3%]) B-ALL. The most common major outpatient blinatumomab site care-delivery challenges included lack of home care companies (75 centers [51.0%]), family distance to treating center (41 centers [27.9%]), and insurance coverage for home care companies (37 centers [25.2%]). There were 67 sites (45.6%) that reported having no home care company options for any patients.
Conclusions and Relevance
In this study, challenges associated with pediatric blinatumomab home care were highly prevalent, with broader implications for health system infrastructure availability. These data highlight a need to plan for clinical implementation strategies alongside the development and testing of novel therapies.
Introduction
B-cell acute lymphoblastic leukemia (B-ALL) is the most common pediatric cancer in the US and accounts for more than 20% of all childhood cancer diagnoses each year.1,2,3 The vast majority of children with cancer (>80%) in the US are treated at a member institution of the Children’s Oncology Group (COG), an international multi-institutional cooperative clinical trials group.4 In 2024, the COG phase 3 randomized clinical trial AALL1731 for National Cancer Institute (NCI) pediatric standard-risk B-ALL (NCT03914625) assessed the efficacy of a novel immunotherapy, blinatumomab, when added to the standard frontline chemotherapy backbone. Children in the experimental arms who received standard B-ALL chemotherapy plus two 28-day cycles of blinatumomab achieved a 3-year disease-free survival of 96% compared with 88% among children in the control arms who received chemotherapy alone.5 These results were so striking that they resulted in early closure of the trial due to superior efficacy of the blinatumomab arm in a planned interim analysis. Along with other trial data from infant and adult populations leading to recent Food and Drug Administration (FDA) approval for blinatumomab as frontline therapy in patients aged 1 month or older, these data established a new standard therapy for most children with B-ALL.6,7,8
However, successful delivery of blinatumomab has unique challenges owing to its administration as a 28-day continuous intravenous infusion. Outpatient delivery can be particularly challenging given the need for associated equipment, requirement for drug bag replacements at variable intervals dependent on institutional clinical capacity and home care service availability (which can be highly variable even within a single institution),9 a care plan for unexpected disconnections, complex insurance reimbursement, and appropriate education for families prior to leaving the hospital.10 Specific subtypes of B-ALL also pose additional care-delivery considerations; these subtypes include infant B-ALL, due to inherent challenges with these patients’ small size and low body weight, and Philadelphia (Ph+) B-ALL, which was not included in the initial FDA approval for use of blinatumomab in frontline therapy.6,10 An earlier survey of COG centers was conducted in 2021, when blinatumomab was offered to only a minority of patients (ie, largely only as an experimental agent on a clinical trial or in more unique relapse settings). This survey revealed wide national variability in the availability of home care services and institutional pharmacy support.11 Given the widely expanded nature of blinatumomab becoming a clinical standard therapy for a much larger number of patients, we hypothesized care delivery challenges in the transition and implementation of this novel immunotherapy from clinical trial to standard clinical practice.
In this study, we sought to characterize the national clinical landscape of pediatric blinatumomab care-delivery practice and challenges in the US from the perspective of treating centers. Secondarily, we explored how care delivery practice and challenges varied by site characteristic.
Methods
This survey study was deemed exempt from review by the Children’s Hospital of Philadelphia institutional review board per 45 CFR §46.104(d) 2(i). Informed consent was obtained electronically; upon accessing the survey link and prior to completing any questions, participants were presented with a description of the study and its purpose, voluntary nature, and confidentiality measures. Moving on to complete the survey after reading this information was explained as implied informed consent for the study. This study is reported following the American Association for Public Opinion Research (AAPOR) reporting guideline for survey studies.
Survey Design and Distribution
A multidisciplinary, multi-institutional team comprised of members from the COG ALL and Cancer Care Delivery Research Committees developed and iteratively revised a survey designed to assess site-level experience with B-ALL, experience with blinatumomab, current care delivery practices, and care delivery challenges. The survey was pilot tested with clinicians from 3 separate institutions and ultimately finalized to include 28 questions. The final, web-based survey was administered via REDCap and sent to all active US COG institutions from February to March 2025 via email to each site’s COG principal investigator and lead COG study coordinator by the central COG Communications office (eAppendix in Supplement 1). We intentionally did not collect data on individual respondent characteristics given that responses were intended to represent site-level practice and respondents were instructed to limit responses to 1 per site. Respondents were encouraged to collaborate with any individuals at their site who could best speak to the institution’s blinatumomab care-delivery practices. Respondents were able to save and return to complete their survey. We sent 2 reminder emails about the survey, and a $50 incentive was offered for survey completion. In the event of duplicate responses for a site, we prioritized answers from the first survey submitted and leveraged the second survey only to impute responses to any questions that were missing answers in the first survey.
Care Delivery Practice by B-ALL Subtype
Sites were asked whether their institutional standard therapy included blinatumomab for each B-ALL subtype. We report results for NCI standard risk (SR)–average (age 1 to <10 years and presenting white blood cell count <50 000/uL [to convert to cells × 109/L, multiply by 0.001]), NCI SR–high, NCI high-risk (age ≥10 years, presenting white blood cell count ≥50 000/uL, or both), infant (age <365 days at diagnosis), and Ph+ B-ALL given that these are risk groups recommended to receive blinatumomab in up-front treatment by most pediatric oncology cooperative groups and National Comprehensive Cancer Network guidelines (eTable 1 in Supplement 1).3 SR-average and SR-high are stratifications of NCI SR defined by the COG risk-stratification schema in AALL1731.5 It is important to note that the level of direct evidence for each subtype varies. Randomized clinical trial results and FDA approval are available for NCI SR-average and NCI SR-high B-ALL from the AALL1731 trial.5 There is no direct evidence for NCI high-risk B-ALL, but it was thought to be generalizable from AALL1731 and was included in the FDA approval. There is encouraging pilot level evidence for infant B-ALL,7 and this was included in the FDA approval. Finally, there is adult-level evidence for Ph+ B-ALL,12,13 but this was not included in the FDA approval.
Care Delivery Challenges
Sites were asked to rank care delivery factors (ie, toxic effects concerns, insurance coverage, lack of home care, institutional challenges, pharmacy staffing, nursing staffing, family preference to decline, family distance, limited family resources, and limited non-English resources) on a Likert scale rating each factor’s impact on their ability to deliver blinatumomab outpatient from 1 (not a problem) to 5 (major problem). This question, along with the previous question regarding care delivery practice by B-ALL subtype, was designed as a required field so that there would be no missing data for survey respondents. For this analysis, we considered a factor to be a major care delivery challenge if an institution rated it 4 or higher. For each care delivery factor, sites were able to submit an optional, open-ended response to give further detail. In this study, we use care delivery factors and challenges as umbrella terms that reflect the multilevel nature of care delivery, including at the patient, center, and system level.
Site Characteristics
We examined site characteristics, including participation in the NCI Community Oncology Research Program (NCORP),14 US census region, site-reported annual pediatric B-ALL volume, prior blinatumomab experience as measured by COG-verified number of AALL1731 enrollments on blinatumomab-containing arms, and prior blinatumomab experience as measured by site-reported number of years using blinatumomab. For NCORP status, US census region, and COG-verified experience with trial-dispensed blinatumomab, data from survey respondents were linked to COG internal data using the unique COG site identifier that sites provided when completing the survey. Site-reported annual pediatric ALL volume was categorized as small volume (0-10 cases of pediatric ALL per year) vs medium volume (11-30 cases) vs large volume (>30 cases).
Statistical Analysis
We compared survey respondents vs nonrespondents by available site characteristics using Pearson χ2 tests or Fisher exact tests, as appropriate. For survey respondents, descriptive frequencies and statistics were tabulated for site characteristics, care delivery practices by B-ALL subtype, and major care delivery challenges. Among centers with no home care company options, we tabulated descriptive frequencies and statistics regarding admission and bag size practices. In the overall cohort, we evaluated whether the site-reported most frequently cited major care delivery challenges (ie, occurring among ≥25% of centers) were associated with site characteristics. Additionally, we examined whether the use of blinatumomab as standard therapy in populations with specific challenges (infant and Ph+ B-ALL) was associated with site characteristics and with reporting of the most frequently cited major care-delivery challenges. These analyses used Pearson χ2 tests or Fisher exact tests, as appropriate, and univariable logistic regression models. Analyses were performed using SAS statistical software version 9.4 (SAS Institute). A 2-sided P < .05 was considered statistically significant for all analyses.
For each care delivery factor, sites were able to include an optional open-ended response to give further detail. After identification of 3 frequently cited major care delivery challenges, a subgroup of authors (D.J.Z., K.O., M.P.B., and S.K.P.) reviewed all open-ended free responses for each of these 3 challenges. After iterative consensus discussions, responses were categorized into higher-order unique perspectives and summarized with supporting quotes.
Results
Site Respondents and Characteristics
The survey was distributed to 195 COG institutions in the US. Overall, 149 sites (76.4% response rate) completed the survey, representing centers in 44 states (states not represented: Washington, Montana, Wyoming, South Dakota, West Virginia, and New Hampshire). There were no statistically significant differences between respondents vs nonrespondents by NCORP status, census region, or prior blinatumomab arm enrollments on AALL1731; 18 survey respondents (12.2%) had more than 10 AALL1731 blinatumomab arm enrollments compared with 2 of 48 survey nonrespondents (4.2%) (P = .09) (eTable 2 in Supplement 1). Of the 149 respondents, 147 sites (98.7%) provided unique COG center identifiers that were successfully linked to the site’s past clinical trial enrollments, forming the analytic cohort. There were 35 site respondents (23.8%) that were NCORP institutions (Table 1). More than 40% of sites (60 sites [40.8%]) were located in the US South census region, while 17% to 21% were located in the Midwest (32 sites [21.8%]), Northeast (30 sites [20.4%]), and West (25 sites [17.0%]) census regions. A similar proportion of sites reported a small (ie, 0-10 cases per year) vs large (ie, >30 cases per year) annual volume of pediatric B-ALL cases (39 sites [26.5%] and 38 sites [25.9%], respectively). With regard to prior blinatumomab experience, most sites had between 1 and 10 prior confirmed blinatumomab-arm enrollments on AALL1731 (114 sites [77.6%]) and at least 5 years of prior experience with blinatumomab (105 sites [71.4%]).
Table 1. Site Characteristics of Survey Respondents.
| Site characteristic | Sites, No. (%) (N = 147) |
|---|---|
| NCORP site | 35 (23.8) |
| Census region | |
| Midwest | 32 (21.8) |
| Northeast | 30 (20.4) |
| South | 60 (40.8) |
| West | 25 (17.0) |
| Annual pediatric ALL volume, new diagnoses/y | |
| Small (0-10) | 39 (26.5) |
| Medium (11-30) | 70 (47.6) |
| Large (>30) | 38 (25.9) |
| No. of AALL1731 enrollments on a blinatumomab-containing arm | |
| 0 | 15 (10.2) |
| 1-3 | 61 (41.5) |
| 4-10 | 53 (36.1) |
| >10 | 18 (12.2) |
| No. of y using blinatumomab | |
| <5.00 | 42 (28.6) |
| 5.00-9.99 | 84 (57.1) |
| ≥10.00 | 21 (14.3) |
Abbreviations: ALL, acute lymphoblastic leukemia; COG, Children’s Oncology Group; NCORP, National Cancer Institute’s Community Oncology Research Program.
Blinatumomab as Institutional Standard Therapy by Pediatric B-ALL Subtype
Most centers reported using blinatumomab as a component of their institutional standard therapy for NCI SR-average (134 sites [91.2%]), NCI SR-high (144 sites [98.0%]), and NCI high-risk (140 sites [95.2%]) B-ALL (Figure). Fewer centers reported using blinatumomab for infant (83 sites [56.5%]) or Ph+ (96 sites [65.3%]) B-ALL.
Figure. Bar Graph of Institutional Standard Therapy Practice for Blinatumomab by Risk Group.
Therapy practice is shown by pediatric Children’s Oncology Group B-cell acute lymphoblastic leukemia (B-ALL) risk group. Standard risk (age 1 to <10 years and presenting white blood cell count <50 000/uL [to convert to cells × 109/L, multiply by 0.001]) and high risk (age ≥10 years, presenting white blood cell count ≥50 000/uL, or both) are definitions used by the National Cancer Institute. Standard risk average and standard risk high are further stratifications of National Cancer Institute standard risk defined by the Children’s Oncology Group risk-stratification schema in AALL1731.5 Ph+ indicates Philadelphia-positive B-ALL.
Major Blinatumomab Care Delivery Challenges
Among all sites, 56 sites (38.1%) reported no major outpatient care-delivery challenges. Among the remaining 91 sites (61.9%), 16 sites (17.6%) reported 1 major challenge, 22 sites (24.2%) reported 2 major challenges, and 53 (58.2%) reported 3 or more major challenges. The most common major outpatient blinatumomab care-delivery challenges affecting 25% or more of site respondents included lack of home care companies (75 sites [51.0%]), family distance to treating center (41 sites [27.9%]), and difficulty with insurance coverage for home care companies (37 sites [25.2%]) (Table 2). There were no statistically significant differences in major outpatient care-delivery challenges by NCORP status, site-reported annual pediatric B-ALL volume, or prior blinatumomab experience. Compared with centers in the South census region, centers in the West census region were more likely to cite lack of home care companies as a major outpatient care-delivery challenge (odds ratio, 3.37; 95% CI, 1.22-9.25) (Table 3).
Table 2. Major Outpatient Care Delivery Challenges.
| Care delivery factor | Sites endorsing factor as a major challenge, No. (%) (N = 147)a |
|---|---|
| Lack of home care companies | 75 (51.0) |
| Family distance to treating center | 41 (27.9) |
| Insurance coverage for home care company | 37 (25.2) |
| Limited family resources | 29 (19.7) |
| Insurance coverage for medication | 25 (17.0) |
| Institutional challenges | 22 (15.0) |
| Limited non–English language resources | 22 (15.0) |
| Pharmacy staffing | 15 (10.2) |
| Nursing staffing | 15 (10.2) |
| Medication-related toxic effects | 14 (9.5) |
| Family declining outpatient treatment | 7 (4.8) |
Sites were asked to rank care delivery factors on a Likert scale rating each factor’s impact on their ability to deliver blinatumomab outpatient from 1 (not a problem) to 5 (major problem). For this analysis, a factor was considered to be a major care delivery challenge if an institution rated it 4 or higher.
Table 3. Prevalence of Major Outpatient Care Delivery Challenges by Site Characteristic.
| Site characteristic | Insurance coverage for home care companya | Lack of home care companiesa | Family distance to treating centera | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Not major challenge, No. (%) (n = 110) | Major challenge, No. (%) (n = 37) | OR (95% CI) | P value | Not major challenge, No. (%) (n = 72) | Major challenge, No. (%) (n = 75) | OR (95% CI) | P value | Not major challenge, No. (%) (n = 106) | Major challenge, No. (%) (n = 41) | OR (95% CI) | P value | |
| NCORP site | ||||||||||||
| No | 83 (75.5) | 29 (78.4) | 1 [Reference] | .72 | 53 (73.6) | 59 (78.7) | 1 [Reference] | .47 | 82 (77.4) | 30 (73.2) | 1 [Reference] | .59 |
| Yes | 27 (24.5) | 8 (21.6) | 0.85 (0.35-2.08) | 19 (26.4) | 16 (21.3) | 0.76 (0.35-1.62) | 24 (22.6) | 11 (26.8) | 1.25 (0.55-2.87) | |||
| Pediatric ALL volume | ||||||||||||
| Small | 31 (28.2) | 8 (21.6) | 1 [Reference] | .63 | 18 (25.0) | 21 (28.0) | 1 [Reference] | .66 | 27 (25.5) | 12 (29.3) | 1 [Reference] | .21 |
| Medium | 50 (45.5) | 20 (54.1) | 1.55 (0.61-3.95) | 37 (51.4) | 33 (44.0) | 0.76 (0.35-1.68) | 55 (51.9) | 15 (36.6) | 0.61 (0.25-1.49) | |||
| Large | 29 (26.4) | 9 (24.3) | 1.20 (0.41-3.54) | 17 (23.6) | 21 (28.0) | 1.06 (0.43-2.60) | 24 (22.6) | 14 (34.1) | 1.31 (0.51-3.38) | |||
| Census region | ||||||||||||
| Midwest | 28 (25.5) | 4 (10.8) | 0.43 (0.13-1.42) | .13 | 18 (25.0) | 14 (18.7) | 1.02 (0.43-2.42) | .08 | 23 (21.7) | 9 (22.0) | 0.78 (0.31-2.00) | .22 |
| Northeast | 22 (20.0) | 8 (21.6) | 1.09 (0.40-2.96) | 13 (18.1) | 17 (22.7) | 1.71 (0.71-4.14) | 26 (24.5) | 4 (9.8) | 0.31 (0.09-1.00) | |||
| South | 45 (40.9) | 15 (40.5) | 1 [Reference] | 34 (47.2) | 26 (34.7) | 1 [Reference] | 40 (37.7) | 20 (48.8) | 1 [Reference] | |||
| West | 15 (13.6) | 10 (27.0) | 2.00 (0.74-5.39) | 7 (9.7) | 18 (24.0) | 3.37 (1.22-9.25) | 17 (16.0) | 8 (19.5) | 0.94 (0.35-2.55) | |||
| No. of AALL1731 enrollments on a blinatumomab-containing arm | ||||||||||||
| 0 | 11 (10.0) | 4 (10.8) | 1.02 (0.29-3.67) | .40 | 5 (6.9) | 10 (13.3) | 1.59 (0.49-5.20) | .18 | 9 (8.5) | 6 (14.6) | 1.88 (0.58-6.10) | .63 |
| 1-3b | 45 (40.9) | 16 (43.2) | 1 [Reference] | 27 (37.5) | 34 (45.3) | 1 [Reference] | 45 (42.5) | 16 (39.0) | 1 [Reference] | |||
| 4-10 | 43 (39.1) | 10 (27.0) | 0.65 (0.27-1.60) | 32 (44.4) | 21 (28.0) | 0.52 (0.25-1.10) | 40 (37.7) | 13 (31.7) | 0.91 (0.39-2.13) | |||
| >10 | 11 (10.0) | 7 (18.9) | 1.79 (0.59-5.41) | 8 (11.1) | 10 (13.3) | 0.99 (0.35-2.86) | 12 (11.3) | 6 (14.6) | 1.41 (0.45-4.37) | |||
| No. of y using blinatumomab | ||||||||||||
| <5 | 31 (28.2) | 11 (29.7) | 1 [Reference] | .97 | 16 (22.2) | 26 (34.7) | 1 [Reference] | .23 | 28 (26.4) | 14 (34.1) | 1 [Reference] | .52 |
| 5-9.99 | 63 (57.3) | 21 (56.8) | 0.88 (0.26-2.98) | 44 (61.1) | 40 (53.3) | 0.46 (0.16-1.34) | 61 (57.5) | 23 (56.1) | 0.47 (0.13-1.67) | |||
| ≥10 | 16 (14.5) | 5 (13.5) | 0.94 (0.40-2.19) | 12 (16.7) | 9 (12.0) | 0.56 (0.26-1.19) | 17 (16.0) | 4 (9.8) | 0.75 (0.34-1.68) | |||
Abbreviations: ALL, acute lymphoblastic leukemia; NCORP, National Cancer Institute’s Community Oncology Research Program; OR, odds ratio.
ORs and CIs were based on univariable logistic regression models. P values were derived from Pearson χ2 or Fisher exact tests, as appropriate.
Given the small number of sites in the first subcategory of the variable of interest, this subcategory was used as the reference group.
There were 67 sites (45.6%) that reported having no home care company options available to provide home care support to their patients. Among these 67 sites, 1 site reported administering blinatumomab in an inpatient setting for most patients, while the remaining sites offered outpatient treatment with bag changes in an outpatient clinic setting. Approximately half of 66 centers offering outpatient treatment (35 centers [53.0%]) reported experience using 7-day bags for patients. The 31 centers not using 7-day bags reported varying factors that impacted the length of bag duration, including limited pharmacist availability (7 centers [22.6%]), limited home care availability (11 centers [35.5%]), limited nursing availability (5 centers [16.1%]), perceived family burden for bag changes (12 centers [38.7%]), cost of medication to the institution (6 centers [19.4%]), and hospital policy (5 centers [16.1%]).
Infant and Ph+ B-ALL Care Delivery by Site Characteristic and Major Care Delivery Challenge
The 64 centers not using blinatumomab for infants, compared with the 83 centers using blinatumomab for infants, were more likely to be small volume (ie, <10 ALL cases per year; 26 centers [40.6%] vs 13 centers [15.7%]; P = .003), have no history of AALL1731 blinatumomab arm enrollments (10 centers [15.6%] vs 5 centers [6.0%]; P = .02), and have less than 5 years of prior experience with blinatumomab (27 centers [42.2%] vs 15 centers [18.1%]; P = .003) (eTable 3 in Supplement 1). Similar patterns were observed for Ph+ ALL. Centers not using blinatumomab for infant ALL were more likely to lack access to home care companies compared with those using it (39 centers [60.9%] vs 36 centers [43.4%]; P = .03); similarly the 51 centers not using blinatumomab for Ph+ ALL compared with 96 centers using it were more likely to lack access to home care companies (35 centers [68.6%] vs 40 centers [41.7%]; P = .002) (eTable 4 in Supplement 1).
Open-Ended Responses Detailing Major Care Delivery Challenges
A total of 116 open-ended responses were reviewed, including 35 responses under insurance coverage for home care, 42 under lack of home care companies, and 39 under family distance from treating center. These were grouped into 4 higher-order perspectives: (1) Even when centers have access to a home care company, availability for patients receiving blinatumomab can be limited by geography (ie, rurality and across states), pediatric age, and insurance coverage (ie, limited partnerships with home care companies); (2) Financial incentives and existing hospital partnerships likely impact home care availability; (3) Centers have developed hospital- or clinic-based work-arounds; (4) There can be notable burden (eg, prolonged inpatient admission, frequent travel, hospital visits for accidental disconnects or other infusion-related issues, responsibility for bag changes, and physical relocation) on the family regardless of home care availability. Supporting example quotes are displayed in Table 4.
Table 4. Perspectives and Supporting Quotes From Open-Ended Responses on Major Outpatient Challenges.
| Perspective | Supporting quotes |
|---|---|
| Even when centers have access to a home care company, availability for patients receiving blinatumomab can be limited by geography (ie, rurality and across states), pediatric age, and insurance coverage (ie, limited partnerships with home care companies). |
|
| Financial incentives and existing hospital partnerships likely impact home care availability. |
|
| Centers have developed hospital- or clinic-based work-arounds. |
|
| There can be notable burden (eg, prolonged inpatient admission, frequent travel, responsibility for bag changes, or physical relocation) on the family regardless of home care availability. |
|
Discussion
In this large national US survey study of centers spanning 44 states, we observed that most COG institutions in the US (>90%) had quickly adapted to blinatumomab as frontline standard therapy for most standard- and high-risk groups of pediatric B-ALL despite its complex care-delivery requirements. However, there is more heterogeneity with regard to specific populations that may have unique care-delivery challenges, including infants and patients with Ph+ B-ALL. Challenges associated with home care availability were highly prevalent and may vary regionally, including availability of companies and nurses and willingness to support blinatumomab care to pediatric patients and insurance coverage for these services if they are available. Notably, 45.6% of centers reported having no home care company options for their patients. These data point toward a health care infrastructure issue with potential implications for family burden with regard to health care use, resource burden on hospitals to design work-arounds, and the capacity to scale other pediatric outpatient care delivery beyond blinatumomab.
Prior work on pediatric home care availability, access, and quality from the perspective of general pediatrics populations similarly raised concerns around the inadequacy of these supports for children in the US.15,16,17 Specifically, studies have pointed toward severe understaffing and inadequate funding to support appropriate training and retention of skilled clinicians.16,18,19 In a large national study of pediatric hospital discharges in 2016, Berry et al20 demonstrated wide state-based variation in the use of home health care. While that study could not comment on whether these variations were attributable to differences in practice preference vs underlying infrastructure, our study builds on these findings with national data over a decade later suggesting that there may be regional variations in home care availability. One potential policy solution includes the American Academy of Pediatrics updated statement in 2025. It emphasized the need for public and private payers to cover pediatric home health care as a medically necessary benefit and advocated for payments to be adequate to attract and retain in-home clinicians.21
To our knowledge, the only prior care-delivery study of blinatumomab was by Withycombe et al,11 which reported practices when blinatumomab was predominantly administered as an experimental agent in the context of clinical trials. In that study, 66% of centers reported no involvement of home health agencies.9,22 While our study potentially reflects some improvement in that statistic, it remains concerning that nearly half of centers continued to struggle with this issue. Our data demonstrate that centers seemed to have responded by creating ways of administering outpatient blinatumomab without home care services, and indeed single-institution reports have begun to surface highlighting the time and resources that hospitals have dedicated to delivering blinatumomab outpatient.9,22 In the absence of home care availability, it remains unclear what various work-arounds represent in terms of family burden given that half of the centers without home care were not using 7-day bags (ie, the longest duration of drug currently available). Ultimately, these models result in patients needing the medication bag changed more than once per week in a clinic or infusion center. This places additional burden on the family to ensure transportation, time off from work or school, and other related indirect time and financial costs.
To our knowledge, this is the first study to examine the treatment and care-delivery landscape for pediatric B-ALL, the most common childhood cancer, after the paradigm-shifting results of the AALL1731 trial. Our study is strengthened by an excellent response rate, with robust representation across the country, including 44 states, a diverse mix of academic and community settings (23.8% NCORP sites), and equal mix of small and large volume centers.
Limitations
This study needs to be interpreted in the context of its limitations. The survey was conducted in February to March 2025 and therefore within the first year of blinatumomab FDA approval for frontline pediatric therapy and formal presentation and publication of AALL1731 results. These data provide an early look at care delivery and implementation challenges, but it is likely that there have been additional changes since the survey was conducted. This is notably a cross-sectional descriptive survey study, and only tests of association have been performed. We additionally do not have data on site rurality or catchment area, which may be important factors to consider. These data are also limited to the perspective of treating centers and cannot specifically comment on patient- or family-level perspectives regarding care delivery–related burden. Future work needs to incorporate these perspectives, and future studies should also examine blinatumomab delivery in other populations and settings. Given the relative novelty of incorporating blinatumomab into frontline therapy, we are unaware of comparative national clinical data regarding the care delivery practice patterns and challenges in adults in the US or for patients in other countries.
Conclusions
In this survey study of pediatric blinatumomab care-delivery practice, we observed an impressive national effort on the part of treating centers to implement into standard therapy a novel immunotherapy with complex delivery issues. Challenges associated with pediatric home care (availability, insurance coverage, and patient distance from the medical center) were highly prevalent and may have broader implications as a health system infrastructure issue. While ongoing efforts will be important to ease outpatient administration of pediatric blinatumomab, these data also highlight a more general need to consider organized clinical implementation strategies alongside the development and testing of novel pediatric cancer therapies.
eAppendix. COG blina survey
eTable 1. Pediatric B-ALL subtype characteristics
eTable 2. Comparison in site characteristics of COG institution survey respondents vs nonrespondents
eTable 3. Blinatumomab as standard therapy for infant and Ph+ ALL by site characteristic
eTable 4. Blinatumomab as standard therapy for infant and Ph+ ALL by major outpatient challenge
Data Sharing Statement
References
- 1.Siegel RL, Kratzer TB, Giaquinto AN, Sung H, Jemal A. Cancer statistics, 2025. CA Cancer J Clin. 2025;75(1):10-45. doi: 10.3322/caac.21871 [DOI] [PMC free article] [PubMed] [Google Scholar]
- 2.Kantarjian H, Pui CH, Jabbour E. Acute lymphocytic leukaemia. Lancet. 2025;406(10506):950-962. doi: 10.1016/S0140-6736(25)00864-5 [DOI] [PubMed] [Google Scholar]
- 3.Inaba H, Teachey D, Annesley C, et al. Pediatric acute lymphoblastic leukemia, version 2.2025, NCCN clinical practice guidelines in oncology. J Natl Compr Canc Netw. 2025;23(2):41-62. doi: 10.6004/jnccn.2025.0006 [DOI] [PubMed] [Google Scholar]
- 4.Lupo PJ, Siegel DA, Schussler NC, et al. Enrollment in children’s oncology group’s clinical trials: population-based linkage with the national childhood cancer registry. J Natl Cancer Inst. 2025;117(9):1868-1874. doi: 10.1093/jnci/djaf134 [DOI] [PMC free article] [PubMed] [Google Scholar]
- 5.Gupta S, Rau RE, Kairalla JA, et al. Blinatumomab in standard-risk B-cell acute lymphoblastic leukemia in children. N Engl J Med. 2025;392(9):875-891. doi: 10.1056/NEJMoa2411680 [DOI] [PMC free article] [PubMed] [Google Scholar]
- 6.Rabik CA, Wang S, Chadda R, et al. FDA approval summary: blinatumomab for the treatment of B-Cell precursor acute lymphoblastic leukemia in the consolidation phase of multiphase chemotherapy. Clin Cancer Res. 2025;31(20):4230-4238. doi: 10.1158/1078-0432.CCR-25-1034 [DOI] [PMC free article] [PubMed] [Google Scholar]
- 7.van der Sluis IM, de Lorenzo P, Kotecha RS, et al. Blinatumomab added to chemotherapy in infant lymphoblastic leukemia. N Engl J Med. 2023;388(17):1572-1581. doi: 10.1056/NEJMoa2214171 [DOI] [PubMed] [Google Scholar]
- 8.Litzow MR, Sun Z, Mattison RJ, et al. Blinatumomab for MRD-negative acute lymphoblastic leukemia in adults. N Engl J Med. 2024;391(4):320-333. doi: 10.1056/NEJMoa2312948 [DOI] [PMC free article] [PubMed] [Google Scholar]
- 9.Oranges K, Duffy KA, McDonough S, et al. Real-world pediatric blinatumomab administration: access to outpatient care delivery and impact of a hospital-dispensed model. Pediatr Blood Cancer. 2026;73(7):e70314. doi: 10.1002/1545-5017.70314 [DOI] [PMC free article] [PubMed] [Google Scholar]
- 10.Zupanec S, Beauchemin M, Rau RE. Navigating B-ALL in the Era of blinatumomab. Am Soc Clin Oncol Educ Book. 2025;45(3):e472778. doi: 10.1200/EDBK-25-472778 [DOI] [PubMed] [Google Scholar]
- 11.Withycombe JS, Kubaney HR, Okada M, et al. Delivery of care for pediatric patients receiving blinatumomab: a Children’s Oncology Group study. Cancer Nurs. 2024;47(6):451-459. doi: 10.1097/NCC.0000000000001309 [DOI] [PMC free article] [PubMed] [Google Scholar]
- 12.Foà R, Bassan R, Vitale A, et al. ; GIMEMA Investigators . Dasatinib–blinatumomab for Ph-positive acute lymphoblastic leukemia in adults. N Engl J Med. 2020;383(17):1613-1623. doi: 10.1056/NEJMoa2016272 [DOI] [PubMed] [Google Scholar]
- 13.Foà R, Bassan R, Elia L, et al. Long-term results of the dasatinib-blinatumomab protocol for adult Philadelphia-positive ALL. J Clin Oncol. 2024;42(8):881-885. doi: 10.1200/JCO.23.01075 [DOI] [PMC free article] [PubMed] [Google Scholar]
- 14.McCaskill-Stevens W, Lyss AP, Good M, Marsland T, Lilenbaum R. The NCI Community Oncology Research Program: what every clinician needs to know. Am Soc Clin Oncol Educ Book. 2013. doi: 10.14694/EdBook_AM.2013.33.e84 [DOI] [PubMed] [Google Scholar]
- 15.Foster CC, Agrawal RK, Davis MM. Home health care for children with medical complexity: workforce gaps, policy, and future directions. Health Aff (Millwood). 2019;38(6):987-993. doi: 10.1377/hlthaff.2018.05531 [DOI] [PubMed] [Google Scholar]
- 16.Simpser E, Hudak ML; Section on Home Care, Committee on Child Health Financing . Financing of pediatric home health care. Pediatrics. 2017;139(3):e20164202. doi: 10.1542/peds.2016-4202 [DOI] [PubMed] [Google Scholar]
- 17.Foster CC, Morales L, Fawcett AJ, Coleman CL. Access and quality of pediatric home healthcare: a systematic review. Home Health Care Manag Pract. 2023;35(4):287-298. doi: 10.1177/10848223231167878 [DOI] [Google Scholar]
- 18.Maynard R, Christensen E, Cady R, et al. Home health care availability and discharge delays in children with medical complexity. Pediatrics. 2019;143(1):e20181951. doi: 10.1542/peds.2018-1951 [DOI] [PubMed] [Google Scholar]
- 19.Boling P. Effects of policy, reimbursement, and regulation on home health care. In: National Research Council (US) Committee on the Role of Human Factors in Home Health Care . The Role of Human Factors in Home Health Care: Workshop Summary. National Academies Press; 2010. Accessed July 27, 2026. https://www.ncbi.nlm.nih.gov/books/NBK210050/ [PubMed] [Google Scholar]
- 20.Berry JG, Hall M, Dumas H, et al. Pediatric hospital discharges to home health and postacute facility care: a national study. JAMA Pediatr. 2016;170(4):326-333. doi: 10.1001/jamapediatrics.2015.4836 [DOI] [PubMed] [Google Scholar]
- 21.Foster CC, Turchi RM; Section on Home Care; Committee on Child Health Financing . Financing of pediatric home health care: policy statement. Pediatrics. 2025;156(6):e2025073624. doi: 10.1542/peds.2025-073624 [DOI] [PubMed] [Google Scholar]
- 22.Parra Del Riego A, Parker K, Thomsen WC, et al. Blinatumomab utilization in pediatric B-cell acute lymphoblastic leukemia: experience from the Mountain West. Pediatr Blood Cancer. 2026;73(7):e70383. doi: 10.1002/1545-5017.70383 [DOI] [PubMed] [Google Scholar]
Associated Data
This section collects any data citations, data availability statements, or supplementary materials included in this article.
Supplementary Materials
eAppendix. COG blina survey
eTable 1. Pediatric B-ALL subtype characteristics
eTable 2. Comparison in site characteristics of COG institution survey respondents vs nonrespondents
eTable 3. Blinatumomab as standard therapy for infant and Ph+ ALL by site characteristic
eTable 4. Blinatumomab as standard therapy for infant and Ph+ ALL by major outpatient challenge
Data Sharing Statement
