Q1 2026 Recap: Hematology News and Updates

The first quarter of 2026 brought meaningful progress across the hematology landscape, with new FDA approvals, updated clinical practice guidelines, and advancing pipeline therapies reshaping how clinicians approach rare and complex blood disorders. From the first-ever pediatric approval for acquired thrombotic thrombocytopenic purpura to landmark guideline updates in aplastic anemia, chronic kidney disease–related anemia, and AL amyloidosis, the quarter underscored a broader shift toward personalized, evidence-based care.

Catch up on the coverage from the HCPLive editorial team with the key developments that defined Q1 2026 in hematology.

FDA Approves Caplacizuman-yhdp (Cavlivi) as First Pediatric aTTP Therapy

On January 5, 2026, the FDA approved caplacizumab-yhdp (Cablivi; Sanofi) for injection to treat pediatric patients aged ≥12 years with acquired thrombotic thrombocytopenic purpura, in combination with plasma exchange and immunosuppressive therapy. The decision builds upon caplacizumab’s initial 2019 approval for adults with aTTP.

The approval was supported by efficacy data from a retrospective chart review of 30 patients aged 2–18 years, in which 80% achieved clinical remission. Notably, the decision makes caplacizumab the first FDA-approved therapy for pediatric aTTP.

FDA Approves 2-Gram Presentation of Fibryga for Acquired Fibrinogen Deficiency

On January 7, 2026, the FDA approved a 2-gram presentation of fibrinogen (human) lyophilized powder for reconstitution (Fibryga) for fibrinogen replacement in patients with acquired fibrinogen deficiency. Fibryga is the first and only virus-inactivated, human plasma–derived fibrinogen concentrate approved for this indication in the US.

FDA Accepts New Drug Application for Rusfertide, Grants Priority Review In Polycythemia Vera

The acceptance of the NDA and grant of Priority Review for rusfertide to treat polycthemia vera was announced by Takeda and Protagonist on March 2, 2026.

According to the release, the decision was based on results from the phase 3 VERIFY, phase 2 REVIEW, and long-term extension THRIVE study, with the Prescription Drug User Fee Act (PDUFA) target action date set for the third quarter of this calendar year.1

ASH Releases 8 Recommendations for Relapsed, Refractory Acute Lymphoblastic Leukemia in Adolescents, Young Adults

New from ASH were 8 clinical recommendations and 1 research-only recommendation to guide management of relapsed or refractory acute lymphoblastic leukemia (ALL) in adolescents and young adults (AYAs), emphasizing immunotherapy approaches.1

The 2026 Guidelines for Management of Relapsed/efractory ALL in AYAs underscore a paradigm shift toward immunotherapies, including blinatumomab and inotuzumab, over chemotherapy while reinforcing the importance of patient-informed, individualized clinical decision-making.

ASH Publishes Guidelines on Diagnosis and Management of Severe and Very Severe Acquired Aplastic Anemia

ASH has released updated guidelines outlining 33 recommendations and 4 good practice statements for the diagnosis and management of severe and very severe acquired aplastic anemia.

The guidance emphasizes improved diagnostic strategies, including expanded genetic testing, and supports personalized treatment approaches such as early use of eltrombopag alongside immunosuppressive therapy and timely transition to second-line options. Aimed at improving outcomes in this rare but life-threatening condition, the recommendations also seek to expand access to appropriate therapies and standardize care across patient populations.

KDIGO Releases New Guideline for Anemia in Chronic Kidney Disease

The 2026 KDIGO guideline update for the management of anemia in chronic kidney disease expands upon the prior 2012 guidance, with updated recommendations and practice points related to iron management, red blood cell (RBC) transfusions, and emerging therapies. The guideline incorporates new evidence intended to support individualized anemia management across the spectrum of CKD care.

ASH Releases New Clinical Practice Guidelines for Diagnosing AL Amyloidosis

Published on January 28, 2026, in Blood Advances, new clinical practice guidelines from ASH outline best practices to support clinicians in recognizing and diagnosing AL amyloidosis, a disease frequently missed or diagnosed late due to its heterogeneous presentation.

The panelists recommend blood and urine testing, including serum immunofixation, urine immunofixation, and serum free light chain assays, when clinical suspicion for AL amyloidosis exists. To confirm diagnosis, the panel supports the use of surrogate biopsies through combined bone marrow biopsy and abdominal fat pad sampling in most cases, while recognizing that target organ biopsies may be appropriate in select clinical scenarios.