Malignant Tumor of the Biliary Tract

Other names
Types
Causes and risk factors
Symptoms and signs
Diagnostic tests
Non-pharmacological treatments (therapies and others)
Drug treatments
Dietary molecular supplements
Immunity-booster / regenerative / stem-cell” drugs
Surgeries
Preventions
When to see doctors (urgent and routine)
What to eat” and “what to avoid
FAQs

A malignant tumor of the biliary tract is a cancer that starts in the thin tubes that carry bile. These tubes are called the bile ducts, and they run inside the liver and outside the liver to the small intestine. When a cancer grows from the cells that line these ducts, doctors call it cholangiocarcinoma or bile duct cancer. The cancer can begin in small ducts inside the liver (intrahepatic), or in larger ducts near the liver hilum (perihilar), or lower down near the pancreas (distal or extrahepatic). All three locations share many features, but their symptoms and tests can differ because of where the blockage happens. Cancer.gov+1

A malignant tumor of the biliary tract is a dangerous growth that starts in the tubes that carry bile from the liver to the small intestine (bile ducts) or in the gallbladder. Doctors group these cancers as cholangiocarcinoma (inside or outside the liver) and gallbladder cancer. These tumors can block the flow of bile, cause jaundice (yellow skin and eyes), pain, weight loss, and infections. Treatment depends on whether surgery can remove the tumor, and whether the cancer has spread. Care usually includes surgery when possible, targeted therapy for gene-driven tumors, immunotherapy for specific biomarkers, chemotherapy, radiation, and procedures to open blocked ducts to relieve symptoms. Cancer.gov+1

In modern medical classification systems, bile duct cancers are grouped into intrahepatic and extrahepatic tumors, with extrahepatic tumors further split into perihilar and distal. This split matters because it guides imaging, biopsy, and surgery planning. Cancer.gov+1

Other names

Doctors and books may use several names for the same disease:

Cholangiocarcinoma (CCA) – the standard medical term for bile duct cancer. Cancer.gov
Bile duct cancer – common plain-language term. Cancer.gov
Intrahepatic cholangiocarcinoma (iCCA) – starts in ducts inside the liver. sysge.org
Perihilar cholangiocarcinoma (pCCA) – starts at the hilum where right and left ducts join; sometimes called Klatskin tumor. NCBI
Distal extrahepatic cholangiocarcinoma (dCCA) – starts in the lower common bile duct near the pancreas. Cancer.gov

Types

1) Intrahepatic cholangiocarcinoma (iCCA).
This type starts in small or medium bile ducts within the liver. It can appear as a mass in the liver and may not cause jaundice at first. It is the second most common primary liver cancer after hepatocellular carcinoma. sysge.org

2) Perihilar cholangiocarcinoma (pCCA).
This type starts at the hepatic hilum, where the right and left hepatic ducts exit the liver and join. It often causes painless jaundice early because it blocks the main outflow of bile. It is sometimes called a Klatskin tumor. NCBI

3) Distal extrahepatic cholangiocarcinoma (dCCA).
This type starts in the lower common bile duct near the pancreas and small intestine. It commonly presents with jaundice and dark urine due to bile flow blockage downstream. Cancer.gov

Pathologists also classify these cancers under the WHO Digestive System Tumours framework using how the cells look under the microscope and (in newer guidance) their molecular features. This ensures consistent diagnosis and reporting across hospitals. PubMed Central+2publications.iarc.who.int+2

Causes and risk factors

Note: A “cause” is often a long-term risk that raises the chance of cancer. Many patients have no known risk factor.

Primary sclerosing cholangitis (PSC).
PSC is a long-lasting disease that scars bile ducts. The chronic inflammation increases cancer risk—far above the general population—especially when PSC occurs with inflammatory bowel disease. NCBI
Liver fluke infection (Opisthorchis viverrini, Clonorchis sinensis).
These parasites live in bile ducts after people eat raw or undercooked freshwater fish in certain regions. Long-term irritation and DNA damage can lead to cancer. PubMed
Hepatolithiasis (bile duct stones inside the liver).
Repeated blockage and infection from stones irritate duct linings and can trigger malignant change over time. PubMed
Choledochal cysts and bile duct malformations.
People born with abnormal dilated ducts have long-standing stasis and inflammation, raising lifetime cancer risk if not corrected. PubMed
Chronic biliary infection and cholangitis.
Recurrent bacterial infection causes cycles of injury and repair in duct tissue, increasing mutation risk. PubMed
Cirrhosis (any cause).
Scarring and regenerative nodules in the liver change the micro-environment of bile ducts and can favor tumor growth, particularly for intrahepatic tumors. PubMed
Chronic viral hepatitis (HBV, HCV).
Long-term hepatitis can lead to cirrhosis and is linked with higher rates of intrahepatic cholangiocarcinoma in some populations. (Association varies by region.) sysge.org
Non-alcoholic fatty liver disease / NASH.
Persistent metabolic liver injury may contribute to risk through chronic inflammation and fibrosis, especially when cirrhosis develops. sysge.org
Alcohol-related liver disease.
Alcohol can cause steatohepatitis and cirrhosis, indirectly raising risk through scarring and inflammation. sysge.org
Gallstones and chronic cholecystitis.
Gallstone disease can disturb bile flow and cause repeated duct irritation, which some studies link to extrahepatic tumors. ResearchGate
Obesity and metabolic syndrome.
Excess body fat and insulin resistance increase systemic inflammation and are associated with higher risk in some cohorts, particularly for extrahepatic disease. ResearchGate
Diabetes mellitus.
Diabetes may add to risk via insulin/IGF pathways and fatty liver changes, though associations are modest and vary between studies. sysge.org
Smoking (tobacco).
Tobacco smoke carcinogens circulate in blood and bile and may slightly increase risk over many years. sysge.org
Exposure to certain chemicals (e.g., thorotrast—historic, nitrosamines).
Some industrial or historical agents have been linked to bile duct cancers decades after exposure. PubMed
Genetic and familial syndromes (rare).
Some inherited conditions that affect bile flow or DNA repair may increase risk, though this is uncommon. sysge.org
Autoimmune cholangiopathies (other than PSC).
Chronic immune-mediated attack on bile ducts leads to inflammation and scarring, which can set the stage for cancerous change. sysge.org
Hepatobiliary-enteric surgical bypass or biliary-enteric anastomosis (long-term).
Abnormal backflow of intestinal contents into bile ducts after some surgeries can cause chronic irritation. sysge.org
Chronic pancreatitis with bile duct involvement.
Inflammation near the distal bile duct may contribute to risk through shared ducts and enzymes. sysge.org
Aging.
Risk increases with age as DNA damage accumulates and repair systems weaken. Cancer.gov
Geographic and environmental factors.
Areas with high liver fluke exposure or certain environmental toxins show higher disease rates, highlighting the role of local risks. PubMed

Symptoms and signs

Painless jaundice (yellow skin and eyes).
When a tumor blocks the main bile duct, bilirubin builds up in the blood. The skin and eyes turn yellow. This is more common in perihilar or distal tumors. Cancer.gov
Dark urine.
Bilirubin spills into the urine and gives it a tea- or cola-like color.
Pale or clay-colored stools.
Little or no bile reaches the intestine, so stool loses its brown color and looks pale.
Itching (pruritus).
Bile salts under the skin cause intense itching, sometimes worse at night.
Right upper belly discomfort or dull pain.
Stretching of the liver capsule or inflamed ducts may cause a heavy ache under the right ribs.
Nausea and poor appetite.
Blocked bile flow and systemic inflammation reduce appetite and can cause queasiness.
Weight loss.
People may lose weight because they eat less and their body uses more energy fighting the cancer.
Fever and chills (if cholangitis occurs).
A blocked duct can get infected. Infection brings fever, chills, and sometimes low blood pressure—this requires urgent care.
Fatigue and weakness.
Cancer-related inflammation and poor nutrition make people tired.
Abdominal bloating.
Poor digestion of fats and gas can cause bloating; advanced cases may also cause fluid build-up (ascites) when the liver is affected.
Back pain.
Pain can spread to the back, especially with distal tumors near the pancreas.
Greasy, floating stools (steatorrhea).
Lack of bile in the gut prevents fat digestion, making stools oily and difficult to flush.
Enlarged gallbladder (Courvoisier sign, noted by doctors).
If the lower bile duct is blocked slowly, the gallbladder can swell without pain. Doctors may feel this on exam.
General itch-related skin changes (scratch marks).
Long-term itching leads to scratch lines and sometimes skin infection.
Bruising more easily.
Poor bile flow reduces absorption of fat-soluble vitamins (including vitamin K), which can lead to easy bruising and nosebleeds over time.

Diagnostic tests

Doctors combine history, exam, blood tests, imaging, and tissue sampling. The best set of tests depends on where the tumor sits and the person’s overall health. International groups such as EASL and the NCI outline these pathways to ensure accurate diagnosis and staging. sysge.org+1

A) Physical examination

Skin and eye inspection for jaundice.
The doctor looks for yellowing, scratch marks from itching, and signs of chronic liver disease.
Abdominal palpation (liver and gallbladder).
The doctor feels for an enlarged liver or a distended, non-tender gallbladder (Courvoisier sign) that can suggest slow extrahepatic blockage.
Right upper quadrant tenderness.
Tenderness may suggest infection (cholangitis) or inflamed ducts, prompting urgent tests.
Signs of chronic liver disease.
Spider nevi, fluid in the belly, small shrunken liver, or muscle wasting can point to advanced liver problems in intrahepatic cases.
Nutritional status and weight trend.
Weight loss and muscle wasting (sarcopenia) are common; documenting them helps guide treatment.

B) “Manual” bedside tests and simple clinical maneuvers

Murphy’s sign (gentle press under right ribs during breath-in).
While classically for gallbladder inflammation, it helps separate gallstone pain from painless jaundice due to a tumor.
Stool color check (patient report or visual).
Pale stools suggest poor bile entry into the intestine, guiding urgent imaging.
Urine dipstick for bilirubin.
A quick clinic test to confirm conjugated bilirubin spilling into urine in obstructive jaundice.
Pruritus severity scoring.
Simple scales (0–10) track itch burden and response to bile drainage.
Fever check with sepsis screening.
Bedside vitals (temperature, blood pressure, heart rate) screen for cholangitis, which requires immediate drainage.

C) Laboratory and pathological tests

Liver function panel (bilirubin, ALP, GGT, AST/ALT).
A cholestatic pattern—high bilirubin, alkaline phosphatase, and GGT—points toward bile duct blockage and triggers imaging. Cancer.gov
Cancer markers: CA 19-9 and CEA.
These blood markers can be elevated in cholangiocarcinoma but are not specific. They help when interpreted with imaging and cholangitis control. sysge.org
Complete blood count and coagulation tests.
Anemia, infection signs, and clotting problems (from vitamin K deficiency or liver dysfunction) are assessed before procedures.
Viral hepatitis testing (HBsAg, anti-HCV).
Detects coexisting hepatitis, which affects risk, prognosis, and treatment planning. sysge.org
Biliary brush cytology (via ERCP) and fluorescence in situ hybridization (FISH).
When strictures are sampled, adding FISH can increase detection of malignant cells. sysge.org
Endoscopic or percutaneous biopsy.
A small tissue piece from the mass or stricture confirms the diagnosis and allows modern molecular tests (e.g., IDH1, FGFR2) that may guide therapy. sysge.org
Cholangioscopy-directed biopsy.
Direct visualization inside the duct with targeted sampling can improve accuracy in difficult strictures. sysge.org
Pathology (WHO classification).
The pathologist reports tumor type, grade, margins, and markers, following WHO Digestive System Tumours standards. PubMed Central

D) Electrodiagnostic tests

No specific electrodiagnostic test diagnoses bile duct cancer.
Nerve or muscle electrical tests (like EMG/NCS) are not used to diagnose biliary tumors. Doctors may use ECG only to check the heart before anesthesia or major surgery. This category is usually “not applicable” for this cancer.
If a paraneoplastic neuropathy is suspected (rare).
In unusual cases with nerve symptoms, EMG/NCS may be ordered to evaluate secondary nerve problems. This does not detect the cancer itself but assesses complications.

E) Imaging tests (at the core of diagnosis and staging)

Right upper quadrant ultrasound.
This is the usual first test. It shows if the bile ducts are dilated and may point to the blockage level. It is quick, widely available, and radiation-free. JACR+1
Contrast-enhanced CT scan of the abdomen.
CT maps the tumor, the ducts, nearby blood vessels, and looks for spread. It helps with staging and surgical planning. ACR ACSearch
MRI of the liver with MRCP (magnetic resonance cholangiopancreatography).
MRI/MRCP gives high-contrast images of bile ducts and surrounding tissue without radiation. It is excellent for locating the blockage and evaluating resectability. ACR ACSearch
ERCP (endoscopic retrograde cholangiopancreatography).
A scope passes through the mouth into the small intestine to inject contrast into the bile duct. Doctors can take brushings/biopsies and place a stent to drain bile. It is now used more for tissue and treatment than for initial diagnosis in many centers. ACR ACSearch
EUS (endoscopic ultrasound) with fine-needle aspiration.
An ultrasound probe on an endoscope can sample nodes and distal duct lesions through the gut wall, helping stage the disease. ACR ACSearch
Percutaneous transhepatic cholangiography (PTC).
A thin needle enters a liver duct through the skin to inject contrast, obtain samples, or place external/internal drains when ERCP is not possible. ACR ACSearch
PET-CT (selected cases).
PET-CT can help look for distant spread or clarify uncertain findings, but it is not always needed. Use depends on local protocols and availability. sysge.org

Imaging societies summarize when to choose US, CT, MRI/MRCP, ERCP, EUS, and PTC based on the patient’s presentation (for example, a painless jaundice pathway). These “appropriateness” statements help standardize care. ACR ACSearch+1

Non-pharmacological treatments (therapies and others)

Biliary stent placement (endoscopic)
Description: An endoscopist passes a flexible camera through the mouth into the small intestine and places a small plastic or metal tube (stent) across the narrowed bile duct to reopen the channel. It is often done during ERCP. Most people go home the same day. The stent may need changes if it clogs.
Purpose: Relieves jaundice and itching, lowers infection risk, improves appetite and strength so other cancer care is possible.
Mechanism: The stent mechanically keeps the duct open so bile drains into the intestine instead of backing up in the liver and blood. Cancer.gov
Percutaneous biliary drainage (PBD)
Description: An interventional radiologist inserts a thin tube through the skin and liver into the blocked duct under imaging guidance. The tube drains bile outside to a bag or internally past the blockage.
Purpose: Quickly reduces bilirubin when endoscopy is not feasible or fails; stabilizes patients before surgery, chemotherapy, or targeted therapy.
Mechanism: Creates a new path for bile flow by bypassing the obstruction. Cancer.gov
Surgical bypass of the bile duct
Description: If the tumor cannot be removed, surgeons may connect the bile duct or gallbladder directly to the intestine to route bile around the blockage.
Purpose: Long-term relief of jaundice and cholangitis when stents are not suitable.
Mechanism: Reroutes bile flow surgically to avoid the blocked segment. NCBI
Curative tumor resection (when possible)
Description: Surgeons remove the tumor with a margin of normal tissue. This may require removing part of the bile duct, nearby liver tissue (hepatectomy), lymph nodes, or the pancreas/duodenum (Whipple).
Purpose: The only potential cure for localized disease.
Mechanism: Physically eliminates all visible cancer with negative margins to reduce recurrence. Mayo Clinic
Liver transplantation (selected cases)
Description: In very carefully chosen perihilar cholangiocarcinoma, some centers use a protocol that combines chemotherapy/radiation followed by liver transplant.
Purpose: Offers a chance for long-term survival in specific, early-stage cases not resectable.
Mechanism: Removes both the tumor and the diseased biliary tree by replacing the liver/bile ducts. Cancer.gov
External beam radiation therapy (EBRT)
Description: High-energy x-rays focus on the tumor site. Treatments are given over several weeks.
Purpose: Controls local tumor growth, relieves pain, and may reduce bleeding or obstruction.
Mechanism: Damages cancer DNA, limiting cancer cell division; normal tissues are protected by planning and image guidance. Cancer.gov
Stereotactic body radiation therapy (SBRT)
Description: Delivers very precise, high-dose radiation in a few sessions to small targets in the liver or bile duct area.
Purpose: Local control for small lesions not suitable for surgery; palliation of pain or obstruction.
Mechanism: High-dose radiation causes targeted cancer cell death with steep dose falloff to spare adjacent organs. Cancer.gov
Brachytherapy (intraluminal radiation)
Description: A radioactive source is placed close to the tumor inside the duct via catheter for a short time.
Purpose: Enhances local control in selected centers when external radiation is insufficient.
Mechanism: Delivers a concentrated radiation dose directly to the tumor lining the duct. Siteman Cancer Center
Photodynamic therapy (PDT)
Description: A light-sensitive drug is given, then a special light is applied endoscopically to the tumor.
Purpose: Palliation to improve biliary drainage and quality of life in unresectable tumors.
Mechanism: Light activates the drug within cancer cells, triggering reactive oxygen species that kill those cells. Cancer.gov
Radiofrequency ablation (RFA) of biliary strictures
Description: Heat is applied endoscopically to tumor tissue within the duct before stenting.
Purpose: Improves stent patency and may decrease tumor burden locally.
Mechanism: Thermal injury destroys tumor tissue lining the duct. Cancer.gov
Transarterial therapies (e.g., Y-90 radioembolization)
Description: Small radioactive beads are injected into the artery feeding the liver tumor.
Purpose: Local control of intrahepatic tumors and symptom relief in selected patients.
Mechanism: Delivers internal radiation via the tumor’s blood supply while sparing most normal liver. Cancer.gov
Nutrition therapy and dietitian support
Description: Individual plans for high-calorie, high-protein foods; small frequent meals; fat-soluble vitamin support if bile flow is poor.
Purpose: Prevent weight loss, preserve muscle, and support healing during treatment.
Mechanism: Replaces calories and nutrients lost to cholestasis, poor appetite, or treatment side effects. Cancer.gov
Pruritus relief without drugs (skin care & bathing)
Description: Cool baths, fragrance-free emollients, loose cotton clothing, nail care to avoid scratching injury.
Purpose: Reduces itching from bile salts in the skin when cholestasis is present.
Mechanism: Cooling and barrier methods lower skin irritation and break the itch–scratch cycle. (Stenting or drainage remains primary.) Cancer.gov
Pain procedures (nerve blocks)
Description: Interventional pain teams can perform celiac plexus blocks for upper abdominal pain.
Purpose: Decreases opioid need and improves daily function.
Mechanism: Local anesthetic ± neurolytic agent interrupts pain signals from upper abdominal organs. Cancer.gov
Exercise and physical therapy (gentle)
Description: Short walks, light resistance, breathing exercises tailored to fatigue level.
Purpose: Maintains strength, reduces fatigue, and supports mood.
Mechanism: Improves mitochondrial capacity, circulation, and muscle mass with low risk. Cancer.gov
Psychosocial support and counseling
Description: Counseling, support groups, caregiver training, and advance-care planning.
Purpose: Reduces anxiety and depression; improves coping and communication.
Mechanism: Evidence-based psycho-oncology tools build resilience and informed decision-making. Cancer.gov
Smoking cessation
Description: Behavioral coaching, nicotine-free strategies, and referral to formal programs.
Purpose: Lowers infection risk, improves wound healing and overall outcomes.
Mechanism: Eliminating tobacco improves oxygenation and immune function. Cancer.gov
Alcohol moderation/abstinence
Description: Structured plans to limit or stop alcohol when liver is stressed.
Purpose: Protects remaining liver function and reduces complications.
Mechanism: Avoids additional liver injury on top of tumor-related cholestasis. Cancer.gov
Vaccination review (e.g., hepatitis B)
Description: Check vaccination history and exposure risks; vaccinate per national guidance if appropriate.
Purpose: Reduces preventable infections that can complicate liver disease and treatment.
Mechanism: Pre-treatment immune priming against key viruses. NCCN
Early palliative care involvement
Description: Symptom management, goals-of-care, caregiver support start at diagnosis, not only end-of-life.
Purpose: Improves quality of life, treatment tolerance, and sometimes survival.
Mechanism: Multidisciplinary team optimizes symptoms (pain, itch, nausea), nutrition, and decision support. Cancer.gov

Drug treatments

Foundational chemo-immunotherapy

Durvalumab (Imfinzi) + gemcitabine/cisplatin
Description: Durvalumab is an anti-PD-L1 antibody. The FDA approved it with gemcitabine/cisplatin for first-line treatment of adults with locally advanced or metastatic biliary tract cancer. It is given intravenously on a schedule with chemotherapy.
Class & Purpose: Immune checkpoint inhibitor used with platinum-based chemotherapy to improve survival vs chemotherapy alone.
Dosage/Time: Given IV; label-directed dosing with gemcitabine/cisplatin cycles; continued until progression or toxicity.
Mechanism: Durvalumab blocks PD-L1 to restore T-cell anticancer activity; gemcitabine/cisplatin damage cancer DNA.
Side effects: Fatigue, nausea, anemia; immune effects (pneumonitis, hepatitis, colitis) require prompt reporting. U.S. Food and Drug Administration+2FDA Access Data+2

FGFR2-altered intrahepatic cholangiocarcinoma

Pemigatinib (Pemazyre)
Description: Oral FGFR inhibitor approved for previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions/rearrangements (requires FDA-approved test).
Class & Purpose: Targeted therapy for FGFR2-driven disease after prior therapy.
Dosage/Time: 13.5 mg orally once daily for 14 days, then 7 days off in 21-day cycles, until progression/toxicity.
Mechanism: Blocks FGFR signaling that drives tumor growth in FGFR2-rearranged tumors.
Side effects: Hyperphosphatemia, nail/skin changes, eye problems; routine labs and eye exams are needed. FDA Access Data+2FDA Access Data+2
Futibatinib (Lytgobi)
Description: Oral, irreversible FGFR2 inhibitor for previously treated, unresectable or metastatic intrahepatic cholangiocarcinoma with FGFR2 fusions or rearrangements.
Class & Purpose: Targeted therapy for FGFR2-altered disease after prior treatment.
Dosage/Time: Per label, oral daily dosing until progression/toxicity.
Mechanism: Irreversibly inhibits FGFR2 signaling to stop growth signals.
Side effects: Hyperphosphatemia, nail/skin issues, ocular effects; careful monitoring required. FDA Access Data+2FDA Access Data+2

IDH1-mutated cholangiocarcinoma

Ivosidenib (Tibsovo)
Description: Oral IDH1 inhibitor approved for previously treated, locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation (requires FDA-approved test).
Class & Purpose: Targeted therapy for IDH1-mutant disease to slow progression after prior therapy.
Dosage/Time: Oral daily dosing per label until progression or toxicity.
Mechanism: Inhibits mutant IDH1 enzyme, lowering 2-hydroxyglutarate and restoring normal cell differentiation.
Side effects: Fatigue, diarrhea, QT prolongation (ECG monitoring), liver enzyme elevations. FDA Access Data

HER2-positive biliary tract cancers

Zanidatamab-hrii (Ziihera)
Description: A bispecific HER2-directed antibody approved (accelerated) for previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer using an FDA-approved test.
Class & Purpose: HER2-targeted biologic for HER2-positive BTC after prior therapy.
Dosage/Time: IV on a fixed schedule per label until progression or toxicity.
Mechanism: Binds two distinct HER2 epitopes, promoting receptor internalization and immune-mediated tumor cell killing.
Side effects: Infusion reactions, diarrhea, fatigue; serious adverse reactions can occur and require monitoring. FDA Access Data+2FDA Access Data+2
Fam-trastuzumab deruxtecan-nxki (Enhertu)
Description: An antibody-drug conjugate with tumor-agnostic accelerated approval for previously treated metastatic HER2-positive solid tumors, which can include BTC when HER2-positive.
Class & Purpose: HER2-directed ADC for patients with high HER2 expression after prior treatment.
Dosage/Time: IV every 3 weeks per label until progression/toxicity.
Mechanism: HER2 antibody delivers a cytotoxic payload inside cancer cells; “bystander effect” may kill nearby tumor cells.
Side effects: Nausea, low blood counts; interstitial lung disease risk requires urgent reporting of cough/shortness of breath. U.S. Food and Drug Administration+1

MSI-H/dMMR solid tumors (tissue-agnostic)

Pembrolizumab (Keytruda)
Description: PD-1 inhibitor with the first tissue-agnostic FDA approval for unresectable/metastatic MSI-H/dMMR solid tumors that progressed after prior therapy and have no satisfactory alternatives; this includes MSI-H/dMMR BTC.
Class & Purpose: Immunotherapy for biomarker-selected patients.
Dosage/Time: 200 mg IV q3w or 400 mg q6w until progression/toxicity per label.
Mechanism: Blocks PD-1 to restore T-cell attack on cancer.
Side effects: Immune-related adverse events (thyroid, liver, lung, skin, colon) need prompt care. U.S. Food and Drug Administration+1

NTRK fusion-positive tumors (tissue-agnostic)

Larotrectinib (Vitrakvi)
Description: TRK inhibitor for adult and pediatric patients with NTRK fusion-positive solid tumors without resistance mutations; applicable to BTC if NTRK fusion is present.
Class & Purpose: Targeted therapy for rare NTRK-driven cancers.
Dosage/Time: Oral dosing by weight/body surface; continue until progression/toxicity.
Mechanism: Inhibits TRK signaling from NTRK gene fusions that drive tumor growth.
Side effects: Dizziness, liver enzyme increases, fatigue; monitoring advised. FDA Access Data+1
Entrectinib (Rozlytrek)
Description: TRK/ROS1 inhibitor with tumor-agnostic approval for NTRK fusion-positive solid tumors (also ROS1 NSCLC).
Class & Purpose: Targeted therapy for NTRK-fused cancers; crosses the blood–brain barrier.
Dosage/Time: 600 mg orally once daily in adults; continue until progression/toxicity.
Mechanism: Blocks TRK signaling and may treat brain metastases due to CNS penetration.
Side effects: Dizziness, weight gain, edema, liver tests; ECG/QT monitoring in some cases. U.S. Food and Drug Administration+1

BRAF V600E mutation (tissue-agnostic)

Dabrafenib + Trametinib
Description: Combination BRAF and MEK inhibitors granted tumor-agnostic approval for unresectable/metastatic solid tumors with BRAF V600E, excluding colorectal cancer; can apply to BTC if this mutation is present.
Class & Purpose: Dual targeted therapy for BRAF-driven tumors.
Dosage/Time: Dabrafenib 150 mg twice daily + trametinib per label dosing, continued until progression/toxicity.
Mechanism: Blocks the MAPK pathway at two steps to prevent resistance and suppress growth.
Side effects: Fever, rash, heart and eye effects; careful monitoring needed. FDA Access Data+1

Notes: Many other anticancer drugs (e.g., gemcitabine, cisplatin, capecitabine, oxaliplatin) are frequently used, but their labels are not BTC-specific. The FDA-listed agents above have BTC-specific or tumor-agnostic approvals that can include BTC when the biomarker is present. Always match therapy to the tumor’s biomarkers and the FDA label. JNCCN

Dietary molecular supplements

No supplement cures biliary tract cancer. Discuss any product with your oncology team to avoid interactions (many targeted drugs and immunotherapies have interaction risks). Evidence is limited and mostly supportive.

Omega-3 fatty acids (fish oil)
Description (150 words): Omega-3s may help maintain weight, reduce inflammation, and support heart health during cancer care. They can improve caloric density of meals for patients with poor appetite. Some studies suggest benefits for treatment-related cachexia, but results are mixed. Doses above typical food intake can raise bleeding risk, especially with procedures or low platelets.
Dosage: Common supplemental range 1–2 g/day EPA+DHA; coordinate with your clinician.
Function/Mechanism: Anti-inflammatory lipid mediators may help modulate cytokines and support nutritional status. Cancer.gov
Vitamin D
Description: Low vitamin D is common in cholestasis. Correcting deficiency supports bone and muscle health.
Dosage: Personalized based on blood levels; often 800–2000 IU/day or as prescribed.
Function/Mechanism: Restores normal calcium balance; supports immunity and muscle function. Cancer.gov
Probiotics
Description: Selected strains may help diarrhea from antibiotics or treatment and support gut comfort.
Dosage: Product-specific; discuss with your team if neutropenic or immunosuppressed.
Function/Mechanism: Modulate gut microbiota and barrier function; reduce some GI symptoms. Cancer.gov
Curcumin (turmeric extract)
Description: Studied for anti-inflammatory properties; human anticancer data are limited and not disease-specific.
Dosage: Standardized extracts vary; potential drug interactions exist.
Function/Mechanism: May inhibit NF-κB and other inflammatory pathways. Cancer.gov
Silymarin (milk thistle)
Description: Popular for “liver support”; robust benefit in cancer is unproven. Possible interactions with drug metabolism.
Dosage: Varies by extract; avoid self-prescribing.
Function/Mechanism: Antioxidant effects; possible hepatoprotective actions in some settings. Cancer.gov
N-Acetylcysteine (NAC)
Description: Antioxidant and glutathione precursor sometimes used for mucus thinning and liver support.
Dosage: Varied; check for interactions.
Function/Mechanism: Replenishes intracellular glutathione; scavenges reactive oxygen species. Cancer.gov
Green tea extract (EGCG)
Description: Antioxidant; excessive doses can injure the liver.
Dosage: Prefer tea; be cautious with high-dose capsules.
Function/Mechanism: Polyphenols may reduce oxidative stress. Cancer.gov
Ginger extract
Description: Helps nausea in some people.
Dosage: Caps or tea; watch for heartburn and interactions.
Function/Mechanism: Acts on gut motility and serotonin receptors related to nausea. Cancer.gov
Selenium (only if deficient)
Description: Trace element for antioxidant enzymes; supplement only if levels are low.
Dosage: Typically 50–200 mcg/day under guidance to avoid toxicity.
Function/Mechanism: Supports glutathione peroxidase and redox balance. Cancer.gov
Resveratrol (food-based focus)
Description: Found in grapes/berries; supplements have uncertain benefit and interaction risks.
Dosage: Prefer food sources.
Function/Mechanism: Antioxidant pathways; not a cancer treatment. Cancer.gov

Immunity-booster / regenerative / stem-cell” drugs

There are no FDA-approved stem-cell drugs to treat biliary tract cancer. The items below describe general contexts where such therapies appear in oncology or supportive care; they are not established BTC treatments. Always avoid unregulated “stem cell” clinics.

Colony-stimulating factors (e.g., G-CSF)
Description (~100 words): Support white blood cell recovery during chemotherapy to lower infection risk; not anticancer.
Dosage: Per weight and regimen.
Function/Mechanism: Stimulates bone marrow to produce neutrophils. Cancer.gov
Erythropoiesis-stimulating agents (ESAs)
Description: Used selectively for anemia from chemotherapy in palliative settings; not for cure-intent use.
Dosage: Label-guided.
Function/Mechanism: Stimulates red blood cell production. Cancer.gov
Autologous hematopoietic stem-cell rescue
Description: Not a treatment for BTC; used in some blood cancers after high-dose chemo.
Dosage: Procedural.
Function/Mechanism: Reinfuses a patient’s own stem cells to restore marrow. Cancer.gov
Allogeneic stem-cell transplant
Description: Not indicated for BTC. Included here to clarify scope; used in specific blood cancers.
Dosage: Procedural.
Function/Mechanism: Replaces marrow with donor stem cells for hematologic diseases. Cancer.gov
Mesenchymal stromal cell infusions (experimental)
Description: Investigational for inflammatory conditions; not approved for BTC, risks include infection and emboli.
Dosage: Experimental only.
Function/Mechanism: Proposed immunomodulation; no proven cancer benefit. Cancer.gov
Regenerative nutrition + exercise program (supportive)
Description: Protein optimization, vitamin repletion, and graded activity to rebuild lean mass during therapy.
Dosage: Dietitian-planned.
Function/Mechanism: Supports muscle repair and immune competence indirectly; not a drug. Cancer.gov

Surgeries

Bile duct resection with reconstruction
Procedure: Removes the diseased duct; reconnects duct to intestine (hepaticojejunostomy).
Why: Potential cure if cancer is localized. Mayo Clinic
Hepatectomy (liver resection)
Procedure: Removes liver segments with intrahepatic bile duct tumor and nearby nodes.
Why: Attempt at complete removal with clean margins. Mayo Clinic
Pancreaticoduodenectomy (Whipple)
Procedure: Removes distal bile duct tumor with part of pancreas, small intestine, and nodes.
Why: Curative intent for distal extrahepatic tumors. Mayo Clinic
Liver transplantation (select protocols)
Procedure: Replaces the liver under strict eligibility and neoadjuvant protocols.
Why: Offers survival benefit for highly selected perihilar tumors. Cancer.gov
Palliative biliary bypass
Procedure: Surgical rerouting of bile around the blockage.
Why: Durable relief of jaundice/infection when stents fail or are not possible. NCBI

Preventions

Avoid or treat liver flukes (safe water/food; deworming where endemic). Parasites raise cholangiocarcinoma risk. Cancer.gov
Get vaccinated for hepatitis B and manage hepatitis B/C early. Chronic hepatitis increases hepatobiliary cancer risks. NCCN
Do not smoke; stop if you do. Smoking harms the liver and bile ducts. Cancer.gov
Limit alcohol. Protects the liver from cirrhosis and inflammation. Cancer.gov
Maintain healthy weight and treat fatty liver. Reduces chronic liver injury. Cancer.gov
Control diabetes and metabolic syndrome. Lowers chronic inflammation. Cancer.gov
Safe handling of industrial chemicals. Reduces toxin exposure linked to liver disease. Cancer.gov
Manage primary sclerosing cholangitis (PSC) with specialist care. Early attention to strictures/infections. Cancer.gov
Prompt care for gallstones and biliary infections. Long-standing irritation can raise risk. Cancer.gov
Regular medical follow-up if you have high-risk conditions. Surveillance allows early detection. Cancer.gov

When to see doctors (urgent and routine)

Immediately if you notice yellow skin/eyes, dark urine, pale stools, fever with chills, severe right-upper abdominal pain, or confusion. These may signal blocked ducts or infection needing emergency care. Cancer.gov
Promptly for unexplained weight loss, poor appetite, persistent nausea, new itching, or new abdominal swelling. These are common warning signs that need evaluation. Cancer.gov
Soon if you have PSC, chronic hepatitis, prior biliary surgery, or liver fluke exposure; you may need imaging and labs. Cancer.gov
Before treatment to discuss biomarker testing (FGFR2, IDH1, HER2, MSI-H/dMMR, NTRK, BRAF). These tests guide modern targeted and immune therapies. JNCCN+1

What to eat” and “what to avoid

Eat more of:

Small, frequent, high-protein meals to fight weight loss and fatigue. Cancer.gov
Lean proteins (fish, eggs, tofu, chicken) to maintain muscle during treatment. Cancer.gov
Soft, easy-to-digest foods (oatmeal, soups, yogurt) on low-appetite days. Cancer.gov
Fruits and vegetables for fiber, vitamins, and hydration. Cancer.gov
Healthy fats (olive oil, avocado, nut butters) for calorie density if fat is tolerated. Cancer.gov

Avoid or limit:

Alcohol (adds liver stress). Cancer.gov
Very fatty or fried foods if they worsen indigestion during cholestasis. Cancer.gov
Raw or undercooked seafood/meat when immune-suppressed. Cancer.gov
High-dose herbal supplements without approval (interaction risks with targeted/immune drugs). Cancer.gov
Sugary drinks and ultra-processed foods that displace needed protein and nutrients. Cancer.gov

FAQs

What is a biliary tract malignancy?
A cancer starting in the bile ducts (cholangiocarcinoma) or gallbladder. It often blocks bile flow and causes jaundice and weight loss. Cancer.gov
How is it staged?
Doctors use scans and sometimes staging surgery to decide if the tumor can be removed (resectable) or not (unresectable/metastatic). NCBI
Is surgery the only cure?
Surgery with clear margins is the main path to cure. Selected perihilar cases may be treated with a transplant protocol. Mayo Clinic+1
What if surgery is not possible?
Treatment aims to control disease and symptoms using stents/drainage, radiation, chemo-immunotherapy, and targeted therapy based on biomarkers. Cancer.gov
Which first-line regimen is common now?
Durvalumab with gemcitabine and cisplatin for many patients with advanced disease. U.S. Food and Drug Administration
Why is biomarker testing essential?
It can reveal FGFR2 fusions, IDH1 mutations, HER2 positivity, MSI-H/dMMR, NTRK fusions, or BRAF V600E—each may unlock specific FDA-approved drugs. FDA Access Data+7FDA Access Data+7FDA Access Data+7
What FGFR drugs exist?
Pemigatinib and futibatinib for FGFR2-rearranged cholangiocarcinoma after prior therapy. FDA Access Data+1
What if my tumor has an IDH1 mutation?
Ivosidenib (oral) may be used after prior therapy. FDA Access Data
What about HER2-positive disease?
Zanidatamab is approved for previously treated HER2-positive BTC; Enhertu has tumor-agnostic approval for previously treated HER2-positive solid tumors. FDA Access Data+1
What if the cancer is MSI-H/dMMR?
Pembrolizumab may be used under its tissue-agnostic approval. U.S. Food and Drug Administration
What if the tumor has an NTRK fusion?
Larotrectinib or entrectinib may be options. FDA Access Data+1
What if it has BRAF V600E?
Dabrafenib plus trametinib can be used under their tumor-agnostic approval (not for colorectal cancer). FDA Access Data
Will supplements cure this cancer?
No. Use supplements only to support nutrition and quality of life, and only with your oncology team’s approval. Cancer.gov
How do doctors relieve jaundice?
Endoscopic stenting or percutaneous drainage opens blocked ducts so bile can flow. Cancer.gov
Where can I read trusted guidelines?
See NCI PDQ and NCCN’s Biliary Tract Cancers (patient and professional versions) for updated recommendations. Cancer.gov+1

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