4.0 Article

Diagnosis, treatment, and follow-up of a case of Wolman disease with hemophagocytic lymphohistiocytosis

Related references

Note: Only part of the references are listed.
Article Pediatrics

Wolman's disease presenting with secondary hemophagocytic lymphohistiocytosis: a case report from Saudi Arabia and literature review

Fahad Alabbas et al.

Summary: There is a potential association between infantile metabolic disorder WD and sHLH, further research is needed to identify appropriate therapeutic approaches.

BMC PEDIATRICS (2021)

Article Genetics & Heredity

Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease

Jane E. Potter et al.

Summary: The study describes a multimodal treatment approach for Wolman disease patients combining ERT, DSR, and HCT. After receiving HCT, patients showed improved disease phenotype and laboratory parameters, particularly in gastrointestinal symptoms. 4 out of 5 patients are alive, demonstrating the significant benefits of HCT in improving the condition of Wolman disease patients.

ORPHANET JOURNAL OF RARE DISEASES (2021)

Review Chemistry, Medicinal

Lysosomal Acid Lipase Deficiency: Therapeutic Options

Gregory M. Pastores et al.

DRUG DESIGN DEVELOPMENT AND THERAPY (2020)

Article Gastroenterology & Hepatology

Early onset lysosomal acid lipase deficiency presenting as secondary hemophagocytic lymphohistiocytosis: Two infants treated with sebelipase alfa

Ermelinda Santos Silva et al.

CLINICS AND RESEARCH IN HEPATOLOGY AND GASTROENTEROLOGY (2018)

Article Medicine, General & Internal

A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency

B. K. Burton et al.

NEW ENGLAND JOURNAL OF MEDICINE (2015)

Article Pediatrics

Wolman disease associated with hemophagocytic lymphohistiocytosis: attempts for an explanation

Roberta Taurisano et al.

EUROPEAN JOURNAL OF PEDIATRICS (2014)

Editorial Material Oncology

Wolman's Disease with Secondary Hemophagocytic Lymphohistiocytosis

F. Rabah et al.

PEDIATRIC HEMATOLOGY AND ONCOLOGY (2014)

Review Oncology

HLH-2004: Diagnostic and therapeutic guidelines for hemophagocytic lymphohistiocytosis

Jan-Inge Henter et al.

PEDIATRIC BLOOD & CANCER (2007)

Article Endocrinology & Metabolism

Primary adrenal insufficiency in children: Twenty years experience at the Sainte-Justine Hospital, Montreal

R Perry et al.

JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM (2005)