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3575 Patient and Health Care Professional Perspectives on Quality of Life and Unmet Needs of People with Chronic Neutropenia: A Survey-Based Assessment

Program: Oral and Poster Abstracts
Session: 904. Outcomes Research—Non-Malignant Conditions: Poster II
Hematology Disease Topics & Pathways:
Research, Clinical Research, immunodeficiency, patient-reported outcomes, Diseases, Immune Disorders, neutropenia, real-world evidence
Sunday, December 11, 2022, 6:00 PM-8:00 PM

Anna Legassie, BA*, Felice Tsui, MPH* and Michele Rhee, MBA, MPH*

X4 Pharmaceuticals, Inc., Boston, MA


Chronic neutropenia (CN) encompasses different blood disorders all characterized by low levels of neutrophils (absolute neutrophil count [ANC] <1500/µL for >3 months), that substantially alters quality of life (QoL) of affected individuals due to disease-related symptoms [James RM. Arch Dis Child. 2006; Donadieu J. Exp Rev Hematol. 2021]. Granulocyte colony-stimulating factor (G-CSF) treatment can improve clinical outcomes, but chronic use is associated with debilitating side effects, such as bone pain, that have negative impact on QoL [Michniacki TF. Blood. 2019; Dale DC. N Engl J Med. 2017; Mehta HM. J Immunol. 2015; Donadieu J. Orphanet J Rare Dis. 2011]. This survey evaluated views of patients/caregivers (P/Cs), and health care professionals (HCPs) on unmet needs, treatment patterns, impact on daily life, and QoL of people living with CN.


Participants were recruited via direct emails to patients with CN and outreach to CN Facebook groups from Savvy Cooperative and X4 Pharmaceuticals. P/Cs (n=22) completed up to 29 survey questions; 13 screening questions captured demographics, neutropenia type, and treatment setting. Patients with chemotherapy-induced neutropenia were ineligible. Additional 16 questions captured information on severity, symptoms, impact on daily life, treatment types, and unmet needs. HCPs (n=7) completed a 13-question survey; 7 screening questions captured data on medical specialty and experience treating neutropenia, and 6 questions captured patient neutropenia type, treatment patterns, and unmet needs. Preliminary results per responses through July 8, 2022, are presented; additional updates are pending upon survey close.


Patients (n=18)/caregivers (n=4) completed a validated survey. Included patients were aged
2 to 65 years (45%, 45–65 years). Neutropenia types were idiopathic (32%), congenital (23%), cyclic (18%), autoimmune (14%), and unsure (14%). Most HCPs (86%) served at large academic institutions, with 57% practicing for >21 years. Medical specialties included pediatric hematology/oncology (57%) and adult/pediatric immunology (57%). Medications prescribed included G-CSF (71%), immunoglobulin (Ig) replacement (43%), and antibiotics (14%). G-CSF (29%) and Ig (29%) had the most difficult-to-manage side effects.

When asked which symptoms most impacted health of a person with CN, P/Cs listed fatigue (77%) and frequent/severe infections (77%) as top symptoms, while HCPs listed mouth sores (71%), dental issues (57%), and frequent/recurrent infections (57%). P/Cs reported long-term neutropenia (90%), emotional stress (68%), and disruption to family/social life and social isolation (64%) as top 3 daily life concerns. When asked what treatment improvements are needed, P/Cs listed administration of medication (64%) and reducing fatigue (36%) at the top; HCPs listed fewer/less frequent severe infections (57%) and administration of medication (57%). Improvements in “administration of medication” included alleviation of the burdens associated with injection/intravenous treatments including injection site reactions.

P/Cs reported G-CSF as the most prescribed treatment (64%). Top issues with G-CSF treatment were bone pain (71%) and fatigue (50%); 50% reported G-CSF dose/frequency adjustments; 45% were due to side effects such as bone pain. Six of 7 HCPs (86%) reported adjusting G-CSF dose/frequency, and 1 (17%) attributed the change to side effects.


Interim survey results show differing perspectives between P/C and HCPs on impact of CN and its treatment on QoL. P/Cs cited fatigue as most impactful symptom and fatigue reduction as a priority treatment need, while HCPs ranked fatigue at the bottom or not at all. Notably, the relationship between fatigue and CN is poorly understood and requires further study. Although both groups reported improvement in medication administration as a need, perceptions of patient experience with current treatment differ. P/Cs more commonly cited side effects as the cause of medication changes. Survey results show considerable impact of CN on the lives of people diagnosed and suggest the existing treatment paradigm is burdensome and insufficiently addresses physical health/QoL needs. Additional studies are required to understand unmet needs of people with CN and reasons behind differing perspectives between P/C and HCP communities.

Disclosures: Legassie: X4 Pharmaceuticals, Inc.: Current Employment; Kiniksa Pharmaceuticals: Consultancy. Tsui: X4 Pharmaceuticals, Inc.: Current Employment. Rhee: X4 Pharmaceuticals, Inc.: Current Employment.

*signifies non-member of ASH