Pomalidomide is an immunomodulatory and antineoplastic agent that is used in the therapy of multiple myeloma. Pomalidomide, like the structurally related agents thalidomide and lenalidomide, is associated with a low rate of serum aminotransferase elevations during therapy and has been implicated in causing rare instances of clinically apparent liver injury which can be severe.


Pomalidomide (pom" a lid' oh mide) is a thalidomide derivative (3-amino-thalidomide) similar to lenalidomide that has potent immunomodulatory and antiangiogenic activity and is used as an antineoplastic agent. The mechanism of action of these agents in the treatment of multiple myeloma is not well defined but may relate to inhibition of tumor necrosis factor (TNF) alpha, a potent proinflammatory cytokine or to stimulation of T and NK cell activity. In vitro and in animal models, pomalidomide had greater antineoplastic activity and was less toxic than thalidomide and lenalidomide, but direct comparisons of these agents in humans have not been done. Pomalidomide was approved for use (combined with dexamethasone) in the United States for refractory multiple myeloma in 2015. It subsequently was given accelerated approval as therapy of Kaposi sarcoma. Pomalidomide has also been used on an experimental basis for myelofibrosis and other myeloproliferative disorders. Pomalidomide is available in capsules of 1, 2, 3 and 4 mg under the brand name Pomalyst. The recommended dose for multiple myeloma is 4 mg daily for 21 days in cycles of 28 days indefinitely or until there is disease progression or intolerance. The dose in Kaposi sarcoma is 5 mg daily for 21 days in cycles of 28 days. Its use is restricted because of teratogenicity and strict adherence to birth control (for both men and women) is required. Side effects of pomalidomide are common and similar to those of thalidomide and lenalidomide and include sedation, dizziness, orthostatic hypotension, neutropenia, thrombocytopenia, anemia, peripheral neuropathy and arterial and venous thromboembolism (for which reason it is usually given with antiplatelet agents such as aspirin or with anticoagulation). Rare but potentially severe adverse events include severe cutaneous reactions, severe neuropathy, secondary malignancies, tumor lysis syndrome and hypersensitivity reactions. Pomalidomide is a teratogen and possible cause of severe birth defects and is available only as a part of a strict Risk Evaluation and Mitigation Strategy (REMS), which requires physician training, written patient informed consent, strict birth control measures, regular monitoring and reporting.


Serum enzyme elevations occur in 1% to 2% of patients taking pomalidomide and are more frequent with higher doses. The enzyme abnormalities are usually mild and self-limited and rarely require drug discontinuation. In addition pomalidomide has been implicated in rare instances of clinically apparent, acute liver injury which can be severe and has been reported to lead to deaths from acute liver failure. However, few of these cases have been published and the clinical features, course and outcome of the typical case of liver injury from pomalidomide have not been defined. Both thalidomide and lenalidomide have been implicated in cases of clinically apparent acute liver injury and the presentation and course of injury is likely to be similar to that caused by pomalidomide. The latency to onset of cases of thalidomide associated liver injury is usually within 1 to 6 weeks of starting the antineoplastic agent. The clinical features vary greatly and can be hepatocellular or cholestatic. Cases of acute liver failure as well as vanishing bile duct syndrome with rapid marked cholestasis and hepatic failure have been described with thalidomide and lenalidomide. Immunoallergic features may be prominent and instances of Stevens Johnson syndrome and toxic epidermal necrolysis with and without liver injury have also been linked to therapy with thalidomide and its derivatives. In most cases, the injury resolves rapidly after therapy is stopped. Monitoring of liver tests at monthly intervals is recommended when using thalidomide and its derivatives, and stopping therapy early may play an important role in preventing severe and fatal outcomes.

Pomalidomide and the thalidomide derivatives have also been implicated in causing an increased risk of graft-vs-host disease after autologous or allogeneic hematopoietic stem cell transplantation (HSCT) as well as after liver, kidney and heart transplantation. There appears to be cross reactivity to this complication among lenalidomide, pomalidomide and thalidomide. Therapy usually requires discontinuation of the antineoplastic agent as well as treatment with high doses of corticosteroids and tacrolimus or sirolimus. Furthermore, hepatic graft-vs-host disease can occasionally present with an acute hepatitis that resembles hepatocellular drug induced liver injury.

Reactivation of hepatitis B has been reported in patients receiving thalidomide, lenalidomide and pomalidomide, but generally only after HSCT and the role of these agents in causing reactivation is not always clear. Indeed, in studies of large numbers of patients treated for multiple myeloma the major risk factor for hepatitis B reactivation was found to be HSCT rather than the specific antineoplastic drugs being used. Indeed, lenalidomide therapy is associated with a reduced risk of reactivation in patients with HSCT (although dexamethasone, thalidomide and bortezomib were not), perhaps because of the immune enhancement typically caused by lenalidomide.

Likelihood score: D (possible cause of clinically apparent liver injury).

Mechanism of Injury

The mechanism of pomalidomide hepatotoxicity is not clear, but it may be related to its activity in reducing TNF-α production, a potent inflammatory cytokine that activates T cells and promotes inflammation, but is also necessary for normal liver regeneration. Alternatively, the injury may be triggered by an intermediate of its metabolism, which is largely mediated by hepatic microsomal enzymes, CYP 1A2 and 3A4.

Outcome and Management

The severity of pomalidomide induced liver injury ranges from transient, asymptomatic elevations in serum enzymes to acute liver injury with jaundice to severe acute liver failure and death. Vanishing bile duct syndrome has been reported with use of thalidomide and lenalidomide but not specifically with pomalidomide. Regular monitoring of liver tests is recommended during pomalidomide therapy. Patients who develop liver test abnormalities should stop therapy and restart treatment only if the abnormalities are transient and not associated with symptoms or jaundice. While not proven, the various thalidomide derivatives are likely to demonstrate cross sensitivity to clinically apparent liver injury.

Drug Class: Antineoplastic Agents, Miscellaneous

Other Related Drugs: Lenalidomide, Thalidomide


Case 1. Acute liver injury with jaundice due to pomalidomide.(1)

A 47 year old African American man with refractory multiple myeloma, who had received multiple courses of therapy and an allogenic hematopoietic cell transplant, developed nausea and rash 3 weeks into a first course of pomalidomide (2 mg daily for 21 days) and dexamethasone (20 mg weekly). Shortly thereafter he was found to be jaundiced and was admitted for evaluation and management. He had no history of liver disease, alcohol abuse or risk factors for viral hepatitis. He had had several bouts of graft-vs-host disease after the hematopoietic cell transplant which had been managed with immunosuppression and extracorporeal photopheresis. On hospital admission, total bilirubin was 16.2 mg/dL (direct 12 mg/dL), ALT 1241 U/L, AST 552 U/L, alkaline phosphatase (Alk P) 337 U/L. The prothrombin time was 22.8 seconds. Tests for hepatitis A, B and C and for EBV and CMV infection were negative. A CT of the abdomen showed no evidence of biliary obstruction. A liver biopsy showed severe hepatocyte necrosis and portal inflammation with lymphocytes, plasma cells and eosinophils. The bile ducts showed reactive changes but no inflammation or loss, and hepatic arteries and veins were normal without endothelitis. The biopsy was considered compatible with severe drug induced liver injury and not suggestive of acute or chronic graft-vs-host disease. Over the next few weeks, liver tests improved and liver tests were only mildly abnormal when he was seen as an outpatient two weeks later.

Key Points

Laboratory Values


This man with advanced refractory multiple myeloma, who had received autologous followed by an allogenic hematopoietic cell transplants, developed jaundice approximately 3 weeks after starting a new chemotherapy regimen of pomalidomide and dexamethasone. The jaundice was initially thought to be due to graft-vs-host disease, but a liver biopsy was more compatible with a severe, acute drug induced hepatocellular injury. Other causes of acute liver injury were appropriately excluded. The time to onset, pattern of serum enzyme elevations, history of preexisting liver disease (graft-vs-host liver injury) and rapid improvement with stopping the implicated agent all resemble the features of drug induced liver injury from thalidomide and lenalidomide. It is not known whether there is cross sensitivity to liver injury among the different thalidomide derivatives. Interestingly, he had previously received thalidomide, evidently without liver injury, so that this episode might represent a reexposure.



Pomalidomide – Pomalyst®


Antineoplastic Agents


Product labeling at DailyMed, National Library of Medicine, NIH






Pauff JM, Gonzalez RS, Sajnani KP, Kassim A, Jagasia M. Post-allograft pomalidomide and reversible hepatotoxicity. Bone Marrow Transplant. 2014;49:1341–2. [PubMed: 24955783]


References updated: 30 August 2022

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    (Among 99 patients with relapsed or refractory multiple myeloma treated with pomalidomide and dexamethasone in a Greek National program, the response rate was 32%, and 80% of patients had at least one adverse event including 8% with hematologic and 5% with non-hematologic grade 3 events, none of which were hepatic).
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    (Among 144 patients with relapsed or refractory multiple myeloma treated with pomalidomide and dexamethasone in a German observational study, the overall response rate was 32% and treated related adverse events arose in 63%, which were severe [grade 3-4] in 36%, none of which were liver related).
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    (Among 28 patients with Kaposi sarcoma [18: 65% with HIV infection] treated with pomalidomide [5 mg daily for 21 of each 28-day cycle], the objective response rate was 71% and adverse events were common including ALT elevations in 25%, all of which were transient, asymptomatic and less than 3 times ULN).