RARE DISEASE
Pediatric systemic lupus erythematosus
Pediatric systemic lupus erythematosus
Pediatric systemic lupus erythematosus
Synonyms: SLE, pediatric onset
Synonyms: SLE, pediatric onset
Synonyms: SLE, pediatric onset
Drug discovery
2
drugs
With orphan designations
Overview
Pediatric systemic lupus erythematosus (SLE) is a severe, chronic autoimmune disease characterized by multisystem inflammation and organ damage, with onset before age 18. It involves immune dysregulation, leading to autoantibody production and inflammatory tissue injury. Compared to adult-onset SLE, pediatric cases are more aggressive, with higher rates of renal, neuropsychiatric, and hematologic involvement [1][6][9].
Burden
Therapies
Hydroxychloroquine as baseline therapy [18], combined with immunosuppressants (mycophenolate, cyclophosphamide) and glucocorticoids [3][13]. Biologics (belimumab, rituximab) and calcineurin inhibitors are used for refractory cases [8][13][18]. Treatment emphasizes early remission and minimizing steroid toxicity [8][18].
Categories: rare neurological diseases, rare renal diseases, rare respiratory diseases, rare systemic and rheumatological diseases, rare systemic or rheumatologic diseases of childhood, rare transplant-related disorders
Research Papers
1,093 drug discovery papers about Pediatric systemic lupus erythematosus, with 2 first-in-class and 6 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
1,093 drug discovery papers about Pediatric systemic lupus erythematosus, with 2 first-in-class and 6 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-07-12 | Theory of mind, empathy, and alexithymia in adolescents with systemic lupus erythematosus: associations with fatigue and internalizing symptoms.
This study aimed to examine theory of mind (ToM), empathy, and alexithymia in adolescents with systemic lupus erythematosus (SLE) compared to typically developing controls (TDC) and to investigate the associations of these socio-emotional measures with fatigue and internalizing symptoms. In this two-center cross-sectional study, 21 adolescents with SLE (median age 16 years; 90.5% female) and 21 age- and sex-matched TDC underwent comprehensive assessment including the Faux Pas Recognition Test, Reading the Mind in the Eyes Test, Empathy and Systemizing Quotient, Alexithymia Questionnaire for Children, Revised Child Anxiety and Depression Scale, Fatigue Severity Scale, Sleep Disturbance Scale for Children, and the Wechsler Intelligence Scale for Children-IV. ToM, empathy, and systemizing scores did not differ between groups, despite a generalized trend toward lower WISC-IV performance in the SLE group and a nominally significant, exploratory deficit in Vocabulary (p = .014, r = .380). Alexithymia, particularly the difficulty identifying feelings dimension, showed strong positive correlations with fatigue and internalizing symptoms in both groups (all r ≥ .74, p < .001). Within the SLE group, an exploratory unadjusted analysis showed that longer disease duration was associated with lower empathy (r = - .592, p = .005); this finding was attenuated after adjustment for age at diagnosis. Fatigue tended to be higher in the SLE group (p = .051) without parallel differences in sleep disturbance. Externally directed social cognition did not differ from controls in stable SLE despite nominally lower vocabulary performance, although the small sample limited power to detect modest differences; internally directed emotional processing, by contrast, emerged as a transdiagnostic correlate of fatigue and internalizing symptoms. In an unadjusted exploratory within-group analysis, longer disease duration was associated with lower empathy. These findings are hypothesis-generating and require replication in larger samples with prespecified multiplicity correction. Key Points • Theory of mind and empathy did not differ from controls in adolescents with stable childhood-onset SLE. • Alexithymia strongly correlates with fatigue and internalizing symptoms in pediatric SLE. • In an unadjusted exploratory analysis, longer disease duration was associated with lower empathy.
2026-07-09 | Case Report: A “visceral vasculitis storm” in Systemic Lupus Erythematosus: simultaneous enteritis, ureteritis, and splenic infarction in a pediatric patient
Background Systemic Lupus Erythematosus (SLE) is a multi-system autoimmune disease. Although lupus enteritis is a recognized manifestation of mesenteric vasculitis, lupus ureteritis is rare. The simultaneous occurrence of both, termed a “visceral vasculitis storm,” poses a significant diagnostic challenge. Clinicians often struggle to differentiate primary ureteritis from secondary mechanical compression caused by intestinal edema (the “monistic” view). Case presentation A 16-year-old girl with a history of transient ptosis and positive ANA/dsDNA results 4 months prior to the current presentation, presented with abdominal pain, fever, and transient dysuria. Initial outpatient CT revealed bilateral hydroureteronephrosis. During hospitalization, the patient's condition deteriorated despite antibiotic therapy. A repeat contrast-enhanced CT (CECT) demonstrated the classic “target sign” in the bowel wall, splenic infarction, and crucially, distinct ureteral wall enhancement. Laboratory findings showed a “high CRP/low PCT” dissociation and a markedly elevated D-Dimer (13.74 μg/mL). The diagnosis was revised to SLE with diffuse visceral vasculitis. The patient responded rapidly to methylprednisolone pulse therapy and IVIG, followed by maintenance therapy with Cyclophosphamide and the dual BLyS/APRIL inhibitor Telitacicept. Conclusion This case highlights that ureteral wall enhancement on CECT can serve as a potentially useful radiologic clue for primary lupus ureteritis. Recognizing the “visceral vasculitis storm” is essential to avoid the pitfalls of diagnostic monism and to initiate aggressive immunosuppressive therapy promptly to prevent irreversible organ damage.
2026-07-03 | Modified treatment protocol for pediatric systemic lupus erythematosus-associated hemophagocytic lymphohistiocytosis with central nervous system involvement: a case report.
Hemophagocytic lymphohistiocytosis (HLH), a severe, life-threatening hyperinflammatory syndrome driven by dysregulated immune activation, is characterized by rapid clinical deterioration and poor outcomes that pose critical challenges for clinical management. Here, we report on a female patient aged 10 years and 3 months who was diagnosed with systemic lupus erythematosus (SLE)-associated hemophagocytic lymphohistiocytosis with central nervous system (CNS) involvement. Treatment with the conventional HLH-94/2004 regimen failed to control disease progression. Subsequently, second-line salvage therapy, consisting of emapalumab in combination with teniposide and dexamethasone, was initiated to account for the CNS involvement. The patient achieved sustained, complete clinical response following use of this modified therapeutic approach. Our findings suggest that this combination regimen may offer a potentially feasible therapeutic alternative for managing patients with SLE-associated HLH accompanied by CNS involvement; however, given the single-case nature of this report, further validation in larger cohorts is needed.
2026-07-12 | Theory of mind, empathy, and alexithymia in adolescents with systemic lupus erythematosus: associations with fatigue and internalizing symptoms.
This study aimed to examine theory of mind (ToM), empathy, and alexithymia in adolescents with systemic lupus erythematosus (SLE) compared to typically developing controls (TDC) and to investigate the associations of these socio-emotional measures with fatigue and internalizing symptoms. In this two-center cross-sectional study, 21 adolescents with SLE (median age 16 years; 90.5% female) and 21 age- and sex-matched TDC underwent comprehensive assessment including the Faux Pas Recognition Test, Reading the Mind in the Eyes Test, Empathy and Systemizing Quotient, Alexithymia Questionnaire for Children, Revised Child Anxiety and Depression Scale, Fatigue Severity Scale, Sleep Disturbance Scale for Children, and the Wechsler Intelligence Scale for Children-IV. ToM, empathy, and systemizing scores did not differ between groups, despite a generalized trend toward lower WISC-IV performance in the SLE group and a nominally significant, exploratory deficit in Vocabulary (p = .014, r = .380). Alexithymia, particularly the difficulty identifying feelings dimension, showed strong positive correlations with fatigue and internalizing symptoms in both groups (all r ≥ .74, p < .001). Within the SLE group, an exploratory unadjusted analysis showed that longer disease duration was associated with lower empathy (r = - .592, p = .005); this finding was attenuated after adjustment for age at diagnosis. Fatigue tended to be higher in the SLE group (p = .051) without parallel differences in sleep disturbance. Externally directed social cognition did not differ from controls in stable SLE despite nominally lower vocabulary performance, although the small sample limited power to detect modest differences; internally directed emotional processing, by contrast, emerged as a transdiagnostic correlate of fatigue and internalizing symptoms. In an unadjusted exploratory within-group analysis, longer disease duration was associated with lower empathy. These findings are hypothesis-generating and require replication in larger samples with prespecified multiplicity correction. Key Points • Theory of mind and empathy did not differ from controls in adolescents with stable childhood-onset SLE. • Alexithymia strongly correlates with fatigue and internalizing symptoms in pediatric SLE. • In an unadjusted exploratory analysis, longer disease duration was associated with lower empathy.
2026-07-09 | Case Report: A “visceral vasculitis storm” in Systemic Lupus Erythematosus: simultaneous enteritis, ureteritis, and splenic infarction in a pediatric patient
Background Systemic Lupus Erythematosus (SLE) is a multi-system autoimmune disease. Although lupus enteritis is a recognized manifestation of mesenteric vasculitis, lupus ureteritis is rare. The simultaneous occurrence of both, termed a “visceral vasculitis storm,” poses a significant diagnostic challenge. Clinicians often struggle to differentiate primary ureteritis from secondary mechanical compression caused by intestinal edema (the “monistic” view). Case presentation A 16-year-old girl with a history of transient ptosis and positive ANA/dsDNA results 4 months prior to the current presentation, presented with abdominal pain, fever, and transient dysuria. Initial outpatient CT revealed bilateral hydroureteronephrosis. During hospitalization, the patient's condition deteriorated despite antibiotic therapy. A repeat contrast-enhanced CT (CECT) demonstrated the classic “target sign” in the bowel wall, splenic infarction, and crucially, distinct ureteral wall enhancement. Laboratory findings showed a “high CRP/low PCT” dissociation and a markedly elevated D-Dimer (13.74 μg/mL). The diagnosis was revised to SLE with diffuse visceral vasculitis. The patient responded rapidly to methylprednisolone pulse therapy and IVIG, followed by maintenance therapy with Cyclophosphamide and the dual BLyS/APRIL inhibitor Telitacicept. Conclusion This case highlights that ureteral wall enhancement on CECT can serve as a potentially useful radiologic clue for primary lupus ureteritis. Recognizing the “visceral vasculitis storm” is essential to avoid the pitfalls of diagnostic monism and to initiate aggressive immunosuppressive therapy promptly to prevent irreversible organ damage.
2026-07-03 | Modified treatment protocol for pediatric systemic lupus erythematosus-associated hemophagocytic lymphohistiocytosis with central nervous system involvement: a case report.
Hemophagocytic lymphohistiocytosis (HLH), a severe, life-threatening hyperinflammatory syndrome driven by dysregulated immune activation, is characterized by rapid clinical deterioration and poor outcomes that pose critical challenges for clinical management. Here, we report on a female patient aged 10 years and 3 months who was diagnosed with systemic lupus erythematosus (SLE)-associated hemophagocytic lymphohistiocytosis with central nervous system (CNS) involvement. Treatment with the conventional HLH-94/2004 regimen failed to control disease progression. Subsequently, second-line salvage therapy, consisting of emapalumab in combination with teniposide and dexamethasone, was initiated to account for the CNS involvement. The patient achieved sustained, complete clinical response following use of this modified therapeutic approach. Our findings suggest that this combination regimen may offer a potentially feasible therapeutic alternative for managing patients with SLE-associated HLH accompanied by CNS involvement; however, given the single-case nature of this report, further validation in larger cohorts is needed.
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Drug Discovery Landscape
2 orphan drug designations for Pediatric systemic lupus erythematosus.
2 orphan drug designations for Pediatric systemic lupus erythematosus.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
baricitinib | small molecules | FDA | 2017-11-02 | — | Eli Lilly and Company |
ustekinumab | antibodies | FDA | 2017-07-18 | — | Janssen Biotech, Inc. |
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