Update on Phase III trial of ULTOMIRIS® (ravulizumab-cwvz) in adults and adolescents with thrombotic microangiopathy after hematopoietic stem cell transplant

PUBLISHED 27 July 2026

High-level results from the ALXN1210-TMA-313 Phase III clinical trial showed that ULTOMIRIS^® (ravulizumab-cwvz) did not achieve statistical significance for the primary endpoint of event-free survival through 26 weeks compared to placebo in adults and adolescents (aged 12 years or older) with thrombotic microangiopathy after hematopoietic stem cell transplant (HSCT-TMA). The primary endpoint was defined as the time from randomization until TMA-related clinical worsening or death, whichever occurred first.

In pediatric patients with HSCT-TMA, the ALXN1210-TMA-314 open-label Phase III trial of ULTOMIRIS demonstrated clinically meaningful overall survival of 87.2% at 26 weeks and 73.4% at 52 weeks, as previously disclosed.^1,2 Alexion, AstraZeneca Rare Disease is advancing regulatory filings for ULTOMIRIS in pediatric patients with HSCT-TMA, based on these results and data from ALX-TMA-502, an external control study, which further supports clinically meaningful benefit on overall survival.^1-3

ULTOMIRIS showed a trend toward treatment benefit in adults and adolescents with HSCT-TMA at 26 weeks compared to placebo. Discussions with health authorities are ongoing regarding the interpretation of these data, including in the context of real-world evidence.³

Following HSCT, a procedure used with increasing frequency to treat some types of cancers and other diseases, the devastating and potentially life-threatening complication of TMA may occur.^4,5 TMA can result in blood clots and damage to the walls of the smallest blood vessels in the circulatory system, which may lead to organ failure and death.⁶ HSCT-TMA is estimated to affect fewer than 6,000 people in the US.⁷

Christopher Dvorak, MD, Professor and Chief of the Division of Pediatric Allergy, Immunology and Bone Marrow Transplantation at UCSF Benioff Children's Hospitals, said: "Children who develop HSCT-TMA have an extremely poor prognosis without targeted treatment. While the scientific understanding of this condition continues to evolve, survival remains a critical measure of clinical benefit. The overall survival results observed in this Phase III pediatric trial are clinically meaningful for this young patient population with significant unmet need and may lead to a targeted treatment option for children facing this serious, post-transplant complication."

Vincent Ho, MD, Director of Clinical Operations, Adult Hematopoietic Stem Cell Transplantation and Institute Physician at Dana-Farber Cancer Institute, Professor of Medicine at Harvard Medical School, said: "HSCT-TMA is a serious complication after stem cell transplant with high rates of associated morbidity and mortality and for which effective therapy remains an area of great unmet need. Conducting a global, randomized trial in this complex, life-threatening, post-transplant condition is exceedingly difficult. While the Phase III trial in adults and adolescents did not meet the primary endpoint, the results add new, important insights to advance the field. Continued evaluation of these data together with results collected from recent, real-world experience will further advance clinical understanding and treatment of this complex transplant complication."

Marc Dunoyer, Chief Executive Officer, Alexion, said: "As the largest, global registrational program conducted across a broad population of patients with HSCT-TMA and the only placebo-controlled trial in this adult population, these trials have demonstrated the potential of ULTOMIRIS to improve survival in pediatric patients with this complex condition. We are moving forward with regulatory filings, with the goal of bringing a new treatment option to pediatric patients with HSCT-TMA and their families as quickly as possible. At the same time, we will continue engagement with global health authorities on potential next steps for the adult indication, as we advance additional analyses in the context of real-world data."

The safety profile observed across the ALXN1210-TMA-313 and ALXN1210-TMA-314 trials was consistent with the known safety profile of ULTOMIRIS and with that seen in patients undergoing HSCT.

Alexion plans to present these data at a forthcoming medical meeting.

ULTOMIRIS has been granted Orphan Drug Designation in the US and Japan for the treatment of HSCT-TMA, as well as Breakthrough Therapy designation by the US FDA for the treatment of pediatric patients with HSCT-TMA.

INDICATIONS & IMPORTANT SAFETY INFORMATION for ULTOMIRIS^® (ravulizumab-cwvz)

INDICATIONS

Paroxysmal Nocturnal Hemoglobinuria (PNH)

ULTOMIRIS is indicated for the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH).

Atypical Hemolytic Uremic Syndrome (aHUS)

ULTOMIRIS is indicated for the treatment of adult and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).

Limitation of Use:

ULTOMIRIS is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).

Generalized Myasthenia Gravis (gMG)

ULTOMIRIS is indicated for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.

Neuromyelitis Optica Spectrum Disorder (NMOSD)

ULTOMIRIS is indicated for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive.

IMPORTANT SAFETY INFORMATION

WARNING: SERIOUS MENINGOCOCCAL INFECTIONS ULTOMIRIS, a complement inhibitor, increases the risk of serious infections caused by Neisseria meningitidis [see Warnings and Precautions (5.1)] Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for meningococcal bacteria (for serogroups A, C, W, Y, and B) at least 2 weeks prior to the first dose of ULTOMIRIS, unless the risks of delaying ULTOMIRIS therapy outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against meningococcal bacteria in patients receiving a complement inhibitor. See Warnings and Precautions (5.1) for additional guidance on the management of the risk of serious infections caused by meningococcal bacteria. Patients receiving ULTOMIRIS are at increased risk for invasive disease caused by Neisseria meningitidis, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious meningococcal infections and evaluate immediately if infection is suspected. Because of the risk of serious meningococcal infections, ULTOMIRIS is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called ULTOMIRIS and SOLIRIS REMS [see Warnings and Precautions (5.2)].

CONTRAINDICATIONS

- Initiation in patients with unresolved serious Neisseria meningitidis infection.

WARNINGS AND PRECAUTIONS

Serious Meningococcal Infections

ULTOMIRIS, a complement inhibitor, increases a patient's susceptibility to serious, life-threatening, or fatal infections caused by meningococcal bacteria (septicemia and/or meningitis) in any serogroup, including non-groupable strains. Life-threatening and fatal meningococcal infections have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors.

Revaccinate patients in accordance with ACIP recommendations considering the duration of ULTOMIRIS therapy. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent ULTOMIRIS therapy is indicated in a patient who is not up to date with meningococcal vaccines according to ACIP recommendations, provide antibacterial drug prophylaxis and administer meningococcal vaccines as soon as possible. Various durations and regimens of antibacterial drug prophylaxis have been considered, but the optimal durations and drug regimens for prophylaxis and their efficacy have not been studied in unvaccinated or vaccinated patients receiving complement inhibitors, including ULTOMIRIS. The benefits and risks of treatment with ULTOMIRIS, as well as those associated with antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by Neisseria meningitidis.

Vaccination does not eliminate the risk of serious meningococcal infections, despite development of antibodies following vaccination.

Closely monitor patients for early signs and symptoms of meningococcal infection and evaluate patients immediately if infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if they occur. Promptly treat known infections. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of ULTOMIRIS in patients who are undergoing treatment for serious meningococcal infection depending on the risks of interrupting treatment in the disease being treated.

ULTOMIRIS and SOLIRIS REMS

Due to the risk of serious meningococcal infections, ULTOMIRIS is available only through a restricted program called ULTOMIRIS and SOLIRIS REMS.

Prescribers must enroll in the REMS, counsel patients about the risk of serious meningococcal infection, provide patients with the REMS educational materials, assess patient vaccination status for meningococcal vaccines (against serogroups A, C, W, Y, and B) and vaccinate if needed according to current ACIP recommendations two weeks prior to the first dose of ULTOMIRIS. Antibacterial drug prophylaxis must be prescribed if treatment must be started urgently, and the patient is not up to date with both meningococcal vaccines according to current ACIP recommendations at least two weeks prior to the first dose of ULTOMIRIS. Patients must receive counseling about the need to receive meningococcal vaccines and to take antibiotics as directed, signs and symptoms of meningococcal infection, and be instructed to carry the Patient Safety Card at all times during and for 8 months following ULTOMIRIS treatment.

Further information is available at https://www.UltSolREMS.com or 1-888-765-4747.

Other Infections

Serious infections with Neisseria species (other than Neisseria meningitidis), including disseminated gonococcal infections, have been reported.

ULTOMIRIS blocks terminal complement activation; therefore, patients may have increased susceptibility to infections, especially with encapsulated bacteria, such as infections caused by Neisseria meningitidis but also Streptococcus pneumoniae, Haemophilus influenzae, and to a lesser extent, Neisseria gonorrhoeae. Children treated with ULTOMIRIS may be at increased risk of developing serious infections due to Streptococcus pneumoniae and Haemophilus influenzae type b (Hib). Administer vaccinations for the prevention of Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) infections according to ACIP recommendations. Patients receiving ULTOMIRIS are at increased risk for infections due to these organisms, even if they develop antibodies following vaccination.

Monitoring Disease Manifestations after ULTOMIRIS Discontinuation

Treatment Discontinuation for PNH

After discontinuing treatment with ULTOMIRIS, closely monitor for signs and symptoms of hemolysis, identified by elevated LDH along with sudden decrease in PNH clone size or hemoglobin, or re-appearance of symptoms such as fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), major adverse vascular event (including thrombosis), dysphagia, or erectile dysfunction. Monitor any patient who discontinues ULTOMIRIS for at least 16 weeks to detect hemolysis and other reactions. If signs and symptoms of hemolysis occur after discontinuation, including elevated LDH, consider restarting treatment with ULTOMIRIS.

Treatment Discontinuation for aHUS

ULTOMIRIS treatment of aHUS should be a minimum duration of 6 months. Due to heterogeneous nature of aHUS events and patient-specific risk factors, treatment duration beyond the initial 6 months should be individualized. There are no specific data on ULTOMIRIS discontinuation. After discontinuing treatment with ULTOMIRIS, patients should be monitored for clinical symptoms and laboratory signs of TMA complications for at least 12 months. TMA complications post-discontinuation can be identified if any of the following is observed: Clinical symptoms of TMA include changes in mental status, seizures, angina, dyspnea, thrombosis or increasing blood pressure. In addition, at least two of the following laboratory signs observed concurrently and results should be confirmed by a second measurement 28 days apart with no interruption: a decrease in platelet count of 25% or more as compared to either baseline or to peak platelet count during ULTOMIRIS treatment; an increase in serum creatinine of 25% or more as compared to baseline or to nadir during ULTOMIRIS treatment; or, an increase in serum LDH of 25% or more as compared to baseline or to nadir during ULTOMIRIS treatment. If TMA complications occur after discontinuation, consider reinitiation of ULTOMIRIS treatment or appropriate organ-specific supportive measures.

Thromboembolic Event Management

The effect of withdrawal of anticoagulant therapy during treatment with ULTOMIRIS has not been established. Treatment should not alter anticoagulant management.

Infusion-Related Reactions

Administration of ULTOMIRIS may result in systemic infusion-related reactions, including anaphylaxis and hypersensitivity reactions. In clinical trials, infusion-related reactions occurred in approximately 1 to 7% of patients, including lower back pain, abdominal pain, muscle spasms, drop or elevation in blood pressure, rigors, limb discomfort, drug hypersensitivity (allergic reaction), and dysgeusia (bad taste). These reactions did not require discontinuation of ULTOMIRIS. If signs of cardiovascular instability or respiratory compromise occur, interrupt ULTOMIRIS and institute appropriate supportive measures.

ADVERSE REACTIONS

Adverse Reactions for PNH

Adverse reactions reported in ≥10% or more of patients with PNH were upper respiratory tract infection and headache. Serious adverse reactions were reported in 15 (6.8%) patients receiving ULTOMIRIS. The serious adverse reactions in patients treated with ULTOMIRIS included hyperthermia and pyrexia. No serious adverse reaction was reported in more than 1 patient treated with ULTOMIRIS. One fatal case of sepsis was identified in a patient treated with ULTOMIRIS. In clinical studies, clinically relevant adverse reactions in 1% of adult patients include infusion-related reactions.

Adverse reactions reported in ≥10% of pediatric patients treated with ULTOMIRIS who were treatment-naïve vs. Eculizumab-experienced were anemia (20% vs. 25%), abdominal pain (0% vs. 38%), constipation (0% vs. 25%), pyrexia (20% vs. 13%), upper respiratory tract infection (20% vs. 75%), pain in extremity (0% vs. 25%), and headache (20% vs. 25%).

Adverse Reactions for aHUS

Most common adverse reactions in patients with aHUS (incidence ≥20%) were upper respiratory tract infection, diarrhea, nausea, vomiting, headache, hypertension and pyrexia. Serious adverse reactions were reported in 42 (57%) patients with aHUS receiving ULTOMIRIS. The most frequent serious adverse reactions reported in more than 2 patients (2.7%) treated with ULTOMIRIS were hypertension, pneumonia and abdominal pain.

Adverse reactions reported in ≥20% of pediatric patients treated with ULTOMIRIS were diarrhea, constipation, vomiting, pyrexia, upper respiratory tract infection, decreased vitamin D, headache, cough, rash, and hypertension.

Adverse Reactions for gMG

Most common adverse reactions in adult patients with gMG (incidence ≥10%) were diarrhea and upper respiratory tract infection. Serious adverse reactions were reported in 20 (23%) of patients treated with ULTOMIRIS and in 14 (16%) patients receiving placebo. The most frequent serious adverse reactions were infections reported in at least 8 (9%) patients treated with ULTOMIRIS and in 4 (4%) patients treated with placebo. Of these infections, one fatal case of COVID-19 pneumonia was identified in a patient treated with ULTOMIRIS and one case of infection led to discontinuation of ULTOMIRIS.

Adverse Reactions for NMOSD

Most common adverse reactions in adult patients with NMOSD (incidence ≥10%) were COVID-19, headache, back pain, arthralgia, and urinary tract infection. Serious adverse reactions were reported in 8 (13.8%) patients with NMOSD receiving ULTOMIRIS.

DRUG INTERACTIONS

Plasma Exchange, Plasmapheresis, and Intravenous Immunoglobulins

Concomitant use of ULTOMIRIS with plasma exchange (PE), plasmapheresis (PP), or intravenous immunoglobulin (IVIg) treatment can reduce serum ravulizumab concentrations and requires a supplemental dose of ULTOMIRIS.

Neonatal Fc Receptor Blockers

Concomitant use of ULTOMIRIS with neonatal Fc receptor (FcRn) blockers (e.g., efgartigimod) may lower systemic exposures and reduce effectiveness of ULTOMIRIS. Closely monitor for reduced effectiveness of ULTOMIRIS.

USE IN SPECIFIC POPULATIONS

Pregnancy Exposure Registry

There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to ULTOMIRIS during pregnancy. Healthcare providers and patients may call 1-833-793-0563 or go to https://www.UltomirisPregnancyStudy.com to enroll in or to obtain information about the registry.

To report SUSPECTED ADVERSE REACTIONS, contact Alexion Pharmaceuticals, Inc. at 1-844-259-6783 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see accompanying full Prescribing Information for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening or fatal meningococcal infections.

Notes

HSCT-TMA

Hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) is a rare, severe and potentially life-threatening type of TMA that occurs following HSCT, a procedure used with increasing frequency to treat some types of cancers and other diseases.^4,5 It is thought that factors associated with HSCT (i.e., conditioning regimens and other complications) induce overactivation and/or dysregulation of the complement system, driving HSCT-TMA. Symptoms of HSCT-TMA can overlap with those of other conditions, which can lead to a misdiagnosis and/or a significant delay in receiving an accurate diagnosis.⁴ HSCT-TMA can be fatal, with one-year survival rates in pediatric patients estimated between 17% and 44% and one-year overall survival rates ranging from approximately 17% to 58% in adults, based upon scientific literature.^8-10

TMAs are a group of severe and potentially life-threatening rare disorders that cause blood clots and damage to the walls of the smallest blood vessels in the circulatory system. These blood clots can cause injury to organs that may lead to organ failure and death.⁶ Signs, symptoms and complications of TMA include organ damage (most commonly in the kidneys), low platelet count, red blood cell abnormalities [i.e. anemia, fragmented red cells (schistocytes)], blood clots and high blood pressure.^11-14

ALXN1210-TMA-313

ALXN1210-TMA-313 is a global, Phase III, randomized, double-blind, placebo-controlled, multicenter trial evaluating the safety and efficacy of ULTOMIRIS in adult and adolescent (aged 12 years or older) participants who have thrombotic microangiopathy (TMA) after hematopoietic stem cell transplant (HSCT). Participants were required to have received HSCT within the past 12 months at the time of screening, as well as a diagnosis of TMA that persisted for at least 72 hours after the initial management of any triggering condition or agent.¹⁵

The dosing regimen was confirmed in an open-label, single-arm period (Stage 1) based on data analysis after 14 participants had completed 21 days of treatment. Patients enrolled thereafter in the study were randomized 1:1 to receive either ULTOMIRIS or placebo administered intravenously for a total of 26 weeks, in addition to best supportive care (Stage 2). Patients in the Stage 2 treatment arm received a loading dose of ULTOMIRIS on Days 1, 5 and 10, followed by regular weight-based maintenance dosing of ULTOMIRIS beginning on Day 15, every eight weeks through the treatment period. Upon completion of the treatment period, participants were followed for an additional 26 weeks.¹⁵

The primary endpoint is event-free survival during the 26-week treatment period, defined as the time from randomization until clinical worsening or death. Key secondary endpoints include overall survival, non-relapse mortality and TMA response criteria. The trial enrolled 146 patients from 18 countries across North America, South America, Europe, Asia and Australia.¹⁵

ALXN1210-TMA-314

ALXN1210-TMA-314 is a global, Phase III, open-label, single-arm, multicenter study evaluating the safety and efficacy of ULTOMIRIS in pediatric patients (aged 28 days to less than 18 years of age) with thrombotic microangiopathy (TMA) after hematopoietic stem cell transplantation (HSCT). Participants were required to have received HSCT within the past 12 months at the time of screening, as well as a diagnosis of TMA that persisted for at least 72 hours after the initial management of any triggering condition or agent.¹⁶

The dosing regimen was confirmed based on data analysis after at least 10 participants had completed 21 days of treatment. All patients received a loading dose of ULTOMIRIS on Days 1, 5 and 10, followed by regular weight-based maintenance dosing of ULTOMIRIS beginning on Day 15 and administered every four weeks for patients weighing less than 20 kg or every eight weeks for patients weighing at least 20 kg through the 26-week treatment period, in addition to best supportive care. The administration of supplemental doses of ULTOMIRIS between maintenance doses was guided by prespecified protocol requirements.¹⁶

The primary endpoint is complete TMA response (defined as a composite measure of hematologic and renal parameters) at 26 weeks. Secondary endpoints include overall survival, non-relapse mortality and TMA response criteria. Upon completion of the treatment period, patients were followed for an additional 26 weeks. The trial enrolled 41 patients from seven countries across North America, Europe and Asia.¹⁶

ALX-TMA-502

ALX-TMA-502 is a global, observational, secondary real-world, retrospective study to assess overall survival in adult and pediatric participants (≥ 28 days of age at the time of diagnosis) diagnosed with HSCT-TMA within 52 weeks after stem cell transplantation. The study included two cohorts (complement inhibitor treatment-naïve participants and participants treated with eculizumab) for adult and pediatric participants, respectively. The HSCT-TMA diagnosis was required to have occurred at least 52 weeks before the eligibility assessment date.³

The study was conducted to provide historical control data and real-world contextualization in the treatment of HSCT-TMA. The primary endpoint is overall survival through 52 weeks after diagnosis, defined as the number of days from the date of TMA diagnosis to the date of death. Secondary endpoints include overall survival at 100 days and 26 weeks after diagnosis; and non-relapse mortality through 100 days, 26 weeks, and 52 weeks after diagnosis. In this study, data were collected from 307 patients in 10 countries across North America, South America, Europe, and Asia.³

ULTOMIRIS^® (ravulizumab-cwvz)

ULTOMIRIS^® (ravulizumab-cwvz), the longest-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body's immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Following a loading dose, ULTOMIRIS is administered intravenously every eight weeks in adults, or every four or eight weeks in pediatric patients (based on body weight).

ULTOMIRIS is approved in the US, EU, Japan and other countries for the treatment of certain adults with paroxysmal nocturnal hemoglobinuria (PNH) and is also approved for certain children with PNH in the US, EU and other countries.

ULTOMIRIS is also approved in the US, EU, Japan and other countries for the treatment of certain adults and children with atypical hemolytic uremic syndrome (aHUS).

Additionally, ULTOMIRIS is approved in the US, EU, Japan, China and other countries for the treatment of certain adults with generalized myasthenia gravis (gMG).

Further, ULTOMIRIS is approved in the US, EU, Japan, China and other countries for the treatment of certain adults with neuromyelitis optica spectrum disorder (NMOSD).

ULTOMIRIS is being assessed as a treatment for additional indications as part of a broad development program.

Alexion
Alexion, AstraZeneca Rare Disease, is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US. For more information, please visit www.alexion.us.

AstraZeneca

AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialization of prescription medicines in Oncology, Rare Disease, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca's innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca-us.com and follow the Company on social media @AstraZeneca.

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References

1. Schoettler M, et al. Ravulizumab plus best supportive care to treat pediatric patients with hematopeietic stem cell transplantation-associated thrombotic microangiopathy: first results from a Phase 3 trial. Presented at European Hematology Association 2025 Congress; 12-15 June 2025; Milan, Italy. Abstract S269.
2. Schoettler M, et al. Ravulizumab plus best supportive care in pediatric patients with hematopoietic stem cell transplantation-associated thrombotic microangiopathy: 52-week results from a Phase 3 trial. Presented at Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Congress; 4-7 February 2026; Salt Lake City, Utah. Presentation 80.
3. ALX-TMA-502 Study. AlexionClinicalTrialTransparency.com. Available here. Accessed July 2026.
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5. Atsuta Y, et al. Continuous and differential improvement in worldwide access to hematopoietic cell transplantation: activity has doubled in a decade with a notable increase in unrelated and non-identical related donors. Haematologica. 2024;109(10):3282-3294.
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7. AstraZeneca Data on File - Epidemiology estimates are composed of a triangulation of different data sources including Data Monitor, Decision Resources Group, Kantar Health, and internal input. Available here. Accessed July 2026.
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10. Vasu S, et al. High incidence of severe TA-TMA increases mortality in adult allogeneic transplant recipients: a prospective MIDAS Consortium study. Blood. 2025;146(5):638-646.
11. Raina R, et al. Atypical hemolytic-uremic syndrome: an update on pathophysiology, diagnosis, and treatment. Ther Apher Dial. 2019;23(1):4-21.
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15. ClinicalTrials.gov. Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant. NCT Identifier: NCT04543591. Available here. Accessed July 2026.
16. ClinicalTrials.gov. Study of Ravulizumab in Pediatric Participants With HSCT-TMA. NCT Identifier: NCT04557735. Available here. Accessed July 2026.