Overview
Leishmaniasis is a group of infectious diseases caused by protozoan parasites of the genus Leishmania, transmitted to humans through the bite of infected female phlebotomine sandflies. It is not a genetic disorder but rather an infectious disease that can affect individuals of any background. The disease manifests in three main clinical forms: visceral leishmaniasis (also known as kala-azar; ICD-10 B55.0), cutaneous leishmaniasis (ICD-10 B55.1), and mucocutaneous leishmaniasis (ICD-10 B55.2). Visceral leishmaniasis is the most severe form and affects internal organs, particularly the spleen, liver, and bone marrow, causing fever, weight loss, hepatosplenomegaly (enlargement of the liver and spleen), pancytopenia (low blood cell counts), and hypergammaglobulinemia. Without treatment, visceral leishmaniasis is almost always fatal. Cutaneous leishmaniasis, the most common form, primarily affects the skin, producing ulcerated lesions at the site of the sandfly bite that can leave disfiguring scars. Mucocutaneous leishmaniasis affects the mucous membranes of the nose, mouth, and throat, potentially causing severe tissue destruction of the face. The immune response of the host plays a significant role in determining disease severity and clinical presentation, and certain host genetic factors (including polymorphisms in genes related to immune function such as those in the HLA system, SLC11A1/NRAMP1, and cytokine genes) may influence susceptibility. Treatment depends on the clinical form, the Leishmania species involved, and geographic location. For visceral leishmaniasis, treatments include liposomal amphotericin B (considered first-line in many regions), miltefosine, and pentavalent antimonial compounds. Cutaneous leishmaniasis may be treated with local therapies, cryotherapy, or systemic antileishmanial drugs depending on severity. Mucocutaneous leishmaniasis typically requires systemic treatment. Leishmaniasis is endemic in tropical and subtropical regions across approximately 90 countries, with an estimated 700,000 to 1 million new cases occurring annually worldwide. The disease disproportionately affects populations living in poverty with limited access to healthcare.
Clinical phenotype terms— hover any for plain English:
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
10 eventsInstitute of Tropical Medicine, Belgium — PHASE3
Universidad Industrial de Santander — PHASE1
Drugs for Neglected Diseases — PHASE2
Institute of Tropical Medicine, Belgium
Universidad de Antioquia — PHASE3
Sichuan Huiyu Pharmaceutical Co., Ltd — NA
Institute of Tropical Medicine, Belgium — PHASE3
Hayder Adnan Fawzi — PHASE2
Hayder Adnan Fawzi — PHASE2
Al-Mustafa University College — PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
1 availableIMPAVIDO
Treatment of visceral leishmaniasis due to Leishmania donovani; cutaneous leishmaniasis due to Leishmania braziliensis, Leishmania guyanensis, and Leishmania panamensis; and mucosal leishmaniasis due …
Treatment of visceral leishmaniasis due to Leishmania donovani; cutaneous leishmaniasis due to Leishmania braziliensis, Leishmania guyanensis, and Leishmania panamensis; and mucosal leishmaniasis due to Leishmania braziliensis.
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Financial Resources
1 resourcesTravel Grants
No travel grants are currently matched to Leishmaniasis.
Community
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1 articlesCaregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
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Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Leishmaniasis
What is Leishmaniasis?
Leishmaniasis is a group of infectious diseases caused by protozoan parasites of the genus Leishmania, transmitted to humans through the bite of infected female phlebotomine sandflies. It is not a genetic disorder but rather an infectious disease that can affect individuals of any background. The disease manifests in three main clinical forms: visceral leishmaniasis (also known as kala-azar; ICD-10 B55.0), cutaneous leishmaniasis (ICD-10 B55.1), and mucocutaneous leishmaniasis (ICD-10 B55.2). Visceral leishmaniasis is the most severe form and affects internal organs, particularly the spleen, l
Are there clinical trials for Leishmaniasis?
Yes — 18 recruiting clinical trials are currently listed for Leishmaniasis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Leishmaniasis?
25 specialists and care centers treating Leishmaniasis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Leishmaniasis?
1 patient support program are currently tracked on UniteRare for Leishmaniasis. See the treatments and support programs sections for copay assistance, eligibility, and contact details.