And as the year is coming to an end I had a look back into 2025 greatest hematology achievements in terms of treatment choice and disease control/prognosis. I believe it was a very good year and one with major breakthroughs. Let me guide you through the greatest milestones of this year.
1) TA-TMA after stem cell transplant: a first approved treatment
- What happened: The FDA approved narsoplimab (Yartemlea) for transplant-associated thrombotic microangiopathy (TA-TMA) in adults and children aged 2 years and older.
- What changed: TA-TMA moved from mostly supportive and off-label approaches to a defined, disease-specific approved therapy.
- Why it matters: TA-TMA can be rapidly life-threatening, with major organ risk (especially kidneys). An approved option supports earlier, standardized treatment.
- What to expect next: Real-world data on best timing, patient selection, and outcomes across transplant centers.
2) Thalassemia: first oral therapy approved for anemia in both alpha and beta forms
- What happened: The FDA expanded approval of mitapivat (Aqvesme) as the first oral treatment for anemia in adults with alpha or beta thalassemia, including transfusion-dependent and non–transfusion-dependent disease.
- What changed: A broad, biology-targeting oral option became available across major thalassemia subtypes.
- Why it matters: For suitable patients, improving hemoglobin and reducing anemia burden can significantly improve daily function and long-term complications.
- What to expect next: Careful monitoring for safety requirements (including liver monitoring) and clearer guidance on who benefits most and how to sequence it.
3) Lymphoma: CAR T-cell therapy reaches marginal zone lymphoma
- What happened: The FDA approved lisocabtagene maraleucel (Breyanzi) for relapsed or refractory marginal zone lymphoma after at least two prior systemic therapies.
- What changed: CAR T-cell therapy expanded into another indolent lymphoma where options can narrow after repeated relapse.
- Why it matters: “Indolent” is not “easy.” Patients can face multiple relapses and cumulative treatment fatigue, so deeper remission options matter.
- What to expect next: Better referral pathways, earlier identification of eligible patients, and more long-term outcome data in real-world practice.
4) Multiple myeloma: another major bispecific antibody milestone
- What happened: The FDA granted accelerated approval to linvoseltamab (Lynozyfic), a BCMA-directed CD3 T-cell engager, for heavily pretreated relapsed or refractory multiple myeloma.
- What changed: The immunotherapy toolbox expanded again for later lines, often with faster access than cellular therapy for many patients.
- Why it matters: Myeloma is increasingly managed through sequencing, each effective new option creates more chances for disease control.
- What to expect next: Refinement of toxicity prevention (CRS, infections), dosing strategies, and best sequencing relative to other BCMA therapies.
5) Sickle cell disease: gene editing moves further into real-world access and younger ages
- What happened: 2025 brought strong emphasis on quality of life outcomes after gene therapy, plus new data in children aged 5 to 11 supporting expansion to younger patients.
- What changed: The focus shifted from “can it work?” to “how do we deliver it safely, monitor long-term, and expand access.”
- Why it matters: Potentially transformative therapies only become true breakthroughs when patients can realistically reach them, with proper infrastructure and follow-up.
- What to expect next: Regulatory submissions to extend eligibility to younger patients, and continued work on access, safety frameworks, and long-term monitoring.
6) AML: menin inhibitors arrive for NPM1-mutated relapsed or refractory disease
- What happened: The FDA approved revumenib (Revuforj) and ziftomenib (Komzifti) for NPM1-mutated relapsed or refractory AML in patients with no satisfactory alternatives (with age criteria differing by label).
- What changed: A high-risk AML subset gained targeted options based on genetics, not just intensity of chemotherapy.
- Why it matters: Relapsed AML is one of the hardest areas in hematology, targeted therapies can open additional paths when standard options are exhausted.
- What to expect next: Combination trials, earlier-line strategies, and clearer sequencing with transplant and other targeted agents.
7) MRD: better measurement that strengthens prognosis and guides decisions
- What happened: Data showcased at ASH 2025 reinforced measurable residual disease (MRD) as an early outcome signal in AML and supported MRD-informed strategy discussions in B-ALL transplant conditioning when MRD is negative by high-sensitivity methods.
- What changed: MRD is increasingly a decision tool, not just a research metric, helping tailor intensity and anticipate relapse risk earlier.
- Why it matters: This is precision hematology in action: avoiding undertreatment and avoiding overtreatment, both of which can harm patients.
- What to expect next: Greater standardization of MRD methods, clearer thresholds for action, and more MRD-guided clinical trials.
8) CLL: stronger evidence for time-limited and MRD-guided targeted therapy
- What happened: CLL17 (NEJM 2025) supported fixed-duration venetoclax-based strategies as noninferior to continuous ibrutinib in a prespecified interim analysis, and an NEJM 2025 report further reinforced MRD-guided targeted strategies.
- What changed: More patients can discuss effective plans that are not automatically indefinite therapy, depending on risk profile and shared decision-making.
- Why it matters: Time-limited strategies can reduce long-term burden while maintaining strong disease control in many patients.
- What to expect next: More refined selection of who should pursue fixed-duration versus continuous strategies, and further integration of MRD into everyday CLL practice.
While 2025 has been a very good year, it is just the beginning of a new era in hematology as we get more patient centered and we try to empower patients more. Looking forward to the upcoming year.
Disclaimer: This article is for informational and educational purposes only and does not constitute medical advice. The breakthroughs and treatments discussed here may not be available in all regions or suitable for every patient. Always consult your hematologist or healthcare provider for diagnosis and treatment decisions.
References
- S. Food and Drug Administration. FDA approves lisocabtagene maraleucel for relapsed or refractory marginal zone lymphoma. Dec 4, 2025. Accessed Dec 31, 2025. U.S. Food and Drug Administration+1
- S. Food and Drug Administration. FDA grants accelerated approval to linvoseltamab-gcpt (Lynozyfic) for relapsed or refractory multiple myeloma. Jul 2, 2025. Accessed Dec 31, 2025. U.S. Food and Drug Administration
- S. Food and Drug Administration. FDA approves revumenib (Revuforj) for relapsed or refractory AML with a susceptible NPM1 mutation. Oct 24, 2025. Accessed Dec 31, 2025. U.S. Food and Drug Administration
- S. Food and Drug Administration. FDA approves ziftomenib (Komzifti) for relapsed or refractory AML with a susceptible NPM1 mutation. Nov 13, 2025. Accessed Dec 31, 2025. U.S. Food and Drug Administration
- US FDA approves Omeros’ Yartemlea for TA-TMA after stem cell transplant. Dec 24, 2025. Accessed Dec 31, 2025. Reuters
- US FDA expands approval of mitapivat (Aqvesme) for anemia in alpha or beta thalassemia. Dec 24, 2025. Accessed Dec 31, 2025. Reuters+1
- NHS England. Revolutionary gene-editing therapy for sickle cell approved for NHS use in England. Jan 31, 2025. Accessed Dec 31, 2025. NHS England
- Exagamglogene autotemcel (Casgevy) for treating severe sickle cell disease in people 12 years and over (TA1044). Feb 26, 2025. Accessed Dec 31, 2025. NICE
- American Society of Hematology. Gene therapy leads to improved quality of life in patients with sickle cell disease and beta thalassemia. Aug 27, 2025. Accessed Dec 31, 2025. American Society of Hematology+1
- Vertex’s gene therapy shows promise in children aged 5 to 11 with sickle cell disease and transfusion-dependent beta thalassemia. Dec 6, 2025. Accessed Dec 31, 2025. Reuters
- American Society of Hematology. Advances in technology help improve safety and access to blood cancer therapies (MRD as early survival indicator in AML). Dec 6, 2025. Accessed Dec 31, 2025. American Society of Hematology
- Al-Sawaf O, et al. Fixed-Duration versus Continuous Treatment for Chronic Lymphocytic Leukemia (CLL17). N Engl J Med. 2025. Accessed Dec 31, 2025. New England Journal of Medicine+1
- Munir T, et al. Measurable Residual Disease–Guided Therapy for Chronic Lymphocytic Leukemia (FLAIR). N Engl J Med. 2025. Accessed Dec 31, 2025. New England Journal of Medicine+1