Hematological disease associated with an acquired peripheral neuropathy

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ORPHA:209016
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1FDA treatments8Treatment centers

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Overview

Hematological disease associated with an acquired peripheral neuropathy is a group classification (Orphanet code 209016) that encompasses several blood disorders in which peripheral nerve damage occurs as a secondary or associated feature. These conditions include hematological malignancies and clonal blood disorders — such as certain lymphomas, leukemias, multiple myeloma, Waldenström macroglobulinemia, and monoclonal gammopathies of undetermined significance (MGUS) — that produce paraproteins (abnormal immunoglobulins) or other factors that damage peripheral nerves. The peripheral neuropathy in these conditions typically manifests as numbness, tingling, weakness, pain, and sensory loss in the extremities, often in a distal symmetric pattern affecting the feet and hands. The nervous system involvement can be caused by direct infiltration of nerves by malignant cells, deposition of abnormal proteins, autoimmune-mediated nerve damage, or as a paraneoplastic phenomenon. The body systems primarily affected include the hematopoietic (blood-forming) system and the peripheral nervous system. Patients may experience fatigue, anemia, recurrent infections, or bleeding tendencies related to the underlying blood disorder, alongside progressive neurological symptoms such as gait instability, muscle wasting, and neuropathic pain. In some cases, the neuropathy may be the presenting symptom that leads to discovery of the underlying hematological condition. Treatment depends on the specific underlying hematological disease and may include chemotherapy, immunotherapy, plasma exchange (plasmapheresis), corticosteroids, intravenous immunoglobulin (IVIG), targeted therapies, or stem cell transplantation. Management of the neuropathy itself may involve pain management, physical therapy, and supportive care. Early identification and treatment of the underlying blood disorder can sometimes stabilize or improve the neuropathy.

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗NORD ↗

FDA & Trial Timeline

1 event
Dec 2009

Wilate: FDA approved

Treatment of spontaneous or trauma-induced bleeding episodes in patients with severe von Willebrand disease (VWD) as well as patients with mild or moderate VWD in whom the use of desmopressin is known or suspected to be ineffective or contraindicated

FDAcompleted

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

1 available

Wilate

von Willebrand Factor/Coagulation Factor VIII Complex (Human)· Octapharma USA, Inc.Orphan Drug
Treatment of spontaneous or trauma-induced bleeding episodes in patients with severe von Willebrand disease (VWD) as well as patients with mild or moderate VWD in whom the use of desmopressin is known

Treatment of spontaneous or trauma-induced bleeding episodes in patients with severe von Willebrand disease (VWD) as well as patients with mild or moderate VWD in whom the use of desmopressin is known or suspected to be ineffective or contraindicated

No actively recruiting trials found for Hematological disease associated with an acquired peripheral neuropathy at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Hematological disease associated with an acquired peripheral neuropathy community →

No specialists are currently listed for Hematological disease associated with an acquired peripheral neuropathy.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Hematological disease associated with an acquired peripheral neuropathy.

Search all travel grants →NORD Financial Assistance ↗

Community

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Common questions about Hematological disease associated with an acquired peripheral neuropathy

What is Hematological disease associated with an acquired peripheral neuropathy?

Hematological disease associated with an acquired peripheral neuropathy is a group classification (Orphanet code 209016) that encompasses several blood disorders in which peripheral nerve damage occurs as a secondary or associated feature. These conditions include hematological malignancies and clonal blood disorders — such as certain lymphomas, leukemias, multiple myeloma, Waldenström macroglobulinemia, and monoclonal gammopathies of undetermined significance (MGUS) — that produce paraproteins (abnormal immunoglobulins) or other factors that damage peripheral nerves. The peripheral neuropathy

How is Hematological disease associated with an acquired peripheral neuropathy inherited?

Hematological disease associated with an acquired peripheral neuropathy follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Hematological disease associated with an acquired peripheral neuropathy typically begin?

Typical onset of Hematological disease associated with an acquired peripheral neuropathy is adult. Age of onset can vary across affected individuals.

What treatment and support options exist for Hematological disease associated with an acquired peripheral neuropathy?

1 patient support program are currently tracked on UniteRare for Hematological disease associated with an acquired peripheral neuropathy. See the treatments and support programs sections for copay assistance, eligibility, and contact details.