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INDICATION
RYTELO® (imetelstat) is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA). See more
Discover patient profiles for eligible RYTELO patients
Consider RYTELO second-line after EMA in ESA-ineligible patients with TD LR-MDS
In a retrospective analysis of health claims,
More than half of transfusion-dependent LR-MDS patients had higher transfusion burden prior to or during second-line treatment1,*
- Transfusion burden increased† with each subsequent line of therapy in patients with LR-MDS1
Explore the case study of a patient with LR-MDS who did not respond to first-line EMA treatment
- RS+
- Transfusion dependence prior to first-line treatment: 6 RBC units over 8 weeks
- sEPO: 250 mU/mL
- Hgb: 7.8 g/dL
- Platelets: 329 X 109/L
- ANC: 3.5 X 109/L
- Erythroid maturation agent (EMA)
- Lack of response to first-line EMA treatment
- Evelyn still required 6 RBC units over the past 8 weeks
- Current sEPO: 506 mU/mL
for LR-MDS, symptomatic anemia in eligible RS+ patients‡ as recommended
by the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®)2
*According to a retrospective analysis of health plan claims between October 2015 and March 2023 for LR-MDS patients based on ICD-10 codes.
†For patients with higher transfusion burden.
‡For certain patients with IPSS-R very low-, low-, or intermediate-risk MDS with non-del(5q) ± other cytogenetic abnormalities and with RS ≥15% (or RS ≥5% with an SF3B1 mutation) with sEPO >500 mU/mL.2
Consider RYTELO second-line in eligible patients with TD LR-MDS, regardless of RS status
~40%-70% of eligible patients may not respond or may lose response to ESAs3
Explore the case study of a patient with LR-MDS who did not respond to first-line ESA treatment
- RS status unknown
- Transfusion dependence prior to first-line treatment: 1 RBC unit weekly every other week for the past 8 weeks
- sEPO: 145 mU/mL
- Hgb: 7.6 g/dL
- Platelets: 165 X 109/L
- ANC: 6.2 X 109/L
- Erythropoietin-stimulating agent (ESA)
- Refractory to ESA treatment after 4 months
- John required 4 RBC units over the past 8 weeks
for LR-MDS, symptomatic anemia in eligible RS- and RS+ patients* as recommended
by the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®)2
*For certain patients with IPSS-R very low-, low-, or intermediate-risk MDS with non-del(5q) ± other cytogenetic abnormalities, with sEPO ≤500 mU/mL, and with either RS <15% (or RS <5% with an SF3B1 mutation) or RS ≥15% (or RS ≥5% with an SF3B1 mutation).2
Consider RYTELO for treatment-naïve patients with TD LR-MDS who are ESA-ineligible
sEPO levels can help predict response to treatment, but not all treatments are indicated for ESA-ineligible patients (sEPO >500 mU/mL)4
- ~10%-20% of patients with LR-MDS may be ineligible for ESAs5
Explore the case study of a newly diagnosed patient with LR-MDS who was ineligible for ESA treatment
- RS-
- Transfusion dependence: 4 RBC units over 8 weeks
- sEPO: 502 mU/mL (ESA-ineligible)
- Hgb: 8.2 g/dL
- Platelets: 200 X 109/L
- ANC: 2.0 X 109/L
- No prior treatment history
- Reported increasing fatigue, weakness, and shortness of breath
- Steve required 4 RBC units over 8 weeks
for LR-MDS, symptomatic anemia in eligible patients with sEPO >500 mU/mL* as recommended
by the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®)2
*For certain patients with IPSS-R very low-, low-, or intermediate-risk MDS with non-del(5q) ± other cytogenetic abnormalities and with RS <15% (or RS <5% with an SF3B1 mutation) with sEPO >500 mU/mL and poor probability to respond to IST.2
ANC, absolute neutrophil count; EMA, erythroid-maturation agent; ESA, erythropoiesis; Hgb, hemoglobin; IPSS-R, Revised International Prognostic Scoring System; IST, immunosuppressive therapy; LR-MDS, lower-risk myelodysplastic syndromes; MDS, myelodysplastic syndromes; NCCN, National Comprehensive Cancer Network; RBC, red blood cell; RS, ring sideroblasts; sEPO, serum erythropoietin; TD, transfusion-dependent.
References: 1. Komrokji RS, Supina D, Navada S, et al. Transfusion independence corresponds with survival in patients with lower-risk myelodysplastic syndrome: real-world evidence from United States insurance claims. Clin Lymphoma Myeloma Leuk. 2025;25(10):e740-e749. 2. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Myelodysplastic Syndromes V.3.2026. © National Comprehensive Cancer Network, Inc. 2026. All rights reserved. Accessed January 12, 2026. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way. 3. Brunner AM, Leitch HA, van de Loosdrecht AA, Bonadies N. Management of patients with lower-risk myelodysplastic syndromes. Blood Cancer J. 2022;12(12):166. 4. Park S, Kelaidi C, Meunier M, et al. The prognostic value of serum erythropoietin in patients with lower-risk myelodysplastic syndromes: a review of the literature and expert opinion. Ann Hematol. 2020;99(1):7-19. 5. Platzbecker U. Treatment of MDS. Blood. 2019;133(10):1096-1107.
INDICATION
RYTELO® (imetelstat) is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA).
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
Thrombocytopenia
RYTELO® can cause thrombocytopenia based on laboratory values. In the clinical trial, new or worsening Grade 3 or 4 decreased platelets occurred in 65% of patients with MDS treated with RYTELO.
Monitor patients with thrombocytopenia for bleeding. Monitor complete blood cell counts prior to initiation of RYTELO, weekly for the first two cycles, prior to each cycle thereafter, and as clinically indicated. Administer platelet transfusions as appropriate. Delay the next cycle and resume at the same or reduced dose, or discontinue as recommended.
Neutropenia
RYTELO can cause neutropenia based on laboratory values. In the clinical trial, new or worsening Grade 3 or 4 decreased neutrophils occurred in 72% of patients with MDS treated with RYTELO.
Monitor patients with Grade 3 or 4 neutropenia for infections, including sepsis. Monitor complete blood cell counts prior to initiation of RYTELO, weekly for the first two cycles, prior to each cycle thereafter, and as clinically indicated. Administer growth factors and anti-infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose, or discontinue as recommended.
Infusion-Related Reactions
RYTELO can cause infusion-related reactions. In the clinical trial, infusion-related reactions occurred in 8% of patients with MDS treated with RYTELO; Grade 3 or 4 infusion-related reactions occurred in 1.7%, including hypertensive crisis (0.8%). The most common infusion-related reaction was headache (4.2%). Infusion-related reactions usually occur during or shortly after the end of the infusion.
Premedicate patients at least 30 minutes prior to infusion with diphenhydramine and hydrocortisone as recommended and monitor patients for at least one hour following the infusion as recommended. Manage symptoms of infusion-related reactions with supportive care and infusion interruptions, decrease infusion rate, or permanently discontinue as recommended.
Embryo-Fetal Toxicity
Based on animal findings, RYTELO can cause embryo-fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with RYTELO and for 1 week after the last dose.
ADVERSE REACTIONS
Serious adverse reactions occurred in 32% of patients who received RYTELO. Serious adverse reactions in >2% of patients included sepsis (4.2%), fracture (3.4%), cardiac failure (2.5%), and hemorrhage (2.5%). Fatal adverse reactions occurred in 0.8% of patients who received RYTELO, including sepsis (0.8%).
Most common adverse reactions (≥10% with a difference between arms of >5% compared to placebo), including laboratory abnormalities, were decreased platelets, decreased white blood cells, decreased neutrophils, increased AST, increased alkaline phosphatase, increased ALT, fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19 infections, and headache.
Please see full Prescribing Information, including Medication Guide.
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