Ferrous Sulfate/Ascorbic Acid

New research re-examines existing practices, identifies gaps and unmet needs ORLANDO, Fla., Dec. 6, 2025 /PRNewswire/ -- Four studies presented at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition spotlight how re-examining existing practices can illuminate new approaches, or the need for new approaches, to improving care and outcomes for patients living with blood disorders. "These four studies all seek in different ways to improve care and outcomes for patients – by addressing care gaps, challenging long-held beliefs, and identifying equity gaps and unmet needs," said Adam Cuker, MD, MS, professor of medicine, chief of the classical hematology section, and director of the Penn Comprehensive Hemophilia and Thrombosis Program at the University of Pennsylvania Perelman School of Medicine in Philadelphia, who moderated the press briefing More Than Medicine: Improving Patient Experience and Access. "These findings speak to the ongoing importance of research that questions existing practices and zeroes in on how to achieve better outcomes for patients." Two studies focus on iron deficiency and iron-deficiency anemia, related disorders that are underdiagnosed and undertreated in certain populations, including reproductive-age women. Iron deficiency occurs when the body's iron stores are too low, and is the most common cause of anemia, when the body has too few red blood cells or those cells don't work properly. The body needs iron to, among other things, form hemoglobin, the component of red blood cells that carries oxygen to the organs and tissues and stores the majority of the body's iron. Findings from the first study indicate that iron deficiency is not only a common problem among pregnant patients, but can be readily addressed with a standardized approach to screening and treatment. Results from this multidisciplinary quality improvement initiative demonstrated significant increases within a year in screening rates, infusions of intravenous (IV) iron, and median hemoglobin levels among pregnant patients. The second study addressed iron-deficiency anemia in patients with a simultaneous acute bacterial infection. Its results challenge the long-time practice of not giving IV iron to these patients because of concern that doing so could worsen the infection. Analysis of data from more than 85,000 patients hospitalized with both iron-deficiency anemia and an acute bacterial infection found that, on the contrary, treatment with IV iron increased patients' hemoglobin levels and significantly improved their survival. The remaining two studies highlight gaps in outcomes and unmet needs for specific groups of patients with blood cancers. The first study – one of the largest to date to compare outcomes for Black and white patients with an aggressive form of leukemia who received treatment in clinical trials supported by the National Cancer Institute (a component of the U.S. National Institutes of Health) – found that Black patients are diagnosed at younger ages and have significantly poorer survival than their white counterparts. Moreover, among patients with a common genetic mutation typically associated with more favorable treatment outcomes, survival for Black patients was less than half that for white patients. The final study sheds light on the financial impact of cancer treatment on the families of children undergoing chemotherapy for a fast-growing type of leukemia. This study found that nearly a third of families struggled to cover basic living expenses or lost 25% or more of their household income at some point during their child's treatment. Quality Improvement Project Results in Increased Screening and Treatment for Iron Deficiency in Pregnancy 595: Implementation of a multidisciplinary quality improvement project standardizing an approach to screening and treating iron deficiency in pregnancy Within a year of initiation, a multidisciplinary project to improve screening and treatment for iron deficiency in pregnancy resulted in a sixfold rise in screening rates for iron deficiency in pregnant patients, a 20-fold rise in the number of intravenous (IV) iron infusions, and a significant improvement in median hemoglobin levels. "Screening rates went from 10% to over 60% within a year," said lead author Richard Godby, MD, a hematologist at the Mayo Clinic in Rochester, Minnesota. "Two-thirds of pregnant patients screened were found to be iron deficient, indicating that this is a very common, but readily fixable problem." Women of child-bearing age are at high risk for iron deficiency. Menstruation and low intake of iron-rich foods are some of the most common causes of iron deficiency among women in this age group, Dr. Godby said. In addition, some commonly used medications, such as proton pump inhibitors, can inhibit the body's ability to absorb iron. The body needs more iron during pregnancy. Iron deficiency and anemia during pregnancy have been associated with adverse outcomes such as fetal growth restriction, premature birth, low birth weight, and compromised development of the fetus's brain and nervous system. Iron deficiency can be diagnosed with a blood test for ferritin, a protein that enables the body to store iron. However, guidelines from the American College of Obstetrics and Gynecology – the professional society that represents most U.S. doctors in this specialty – currently recommend iron deficiency screening only for pregnant women with anemia, which they define as a hemoglobin level below 11 g/dL in the first or third trimester. Dr. Godby and his colleagues worked with a multidisciplinary team at the Mayo Clinic to develop and implement a quality improvement project aimed at standardizing the screening and treatment of iron deficiency in pregnancy. They added ferritin testing to the list of recommended lab tests that patients typically undergo at eight to 12 weeks of pregnancy and again at 24 to 28 weeks. If patients had low ferritin levels at eight to 12 weeks, their teams offered to prescribe oral iron supplements. If patients' ferritin was low at 24 to 28 weeks, the teams offered them an IV infusion of iron dextran. To measure the project's results, the research team compared changes after project implementation between the two cohorts of patients – one treated before implementation (2,097 pregnancies; the Before cohort) and one treated a year later, after implementation (2,429 pregnancies; the After cohort). Results showed that, in the Before cohort, just 10% of patients underwent ferritin testing, compared with 63% in the After cohort. Among those tested, 66% in the Before cohort and 69% in the After cohort were iron deficient. Just 0.9% of patients in the Before cohort received IV iron dextran infusions, compared with 21% in the After cohort. Among patients who received IV iron infusions, the median hemoglobin level improved from 10.7 to 11.8 g/dL. Patients whose hemoglobin level was 12 g/dL at study entry (above the cutoff of 11 g/dL to be considered anemic according to current guidelines from the American College of Obstetricians and Gynecologists) saw an increase to 12.8 g/dL. "These findings suggest reassessing the threshold for diagnosing anemia and screening for iron deficiency in pregnancy," Dr. Godby said. Before the project, 3.1% of pregnancies required a blood transfusion during hospitalization for delivery, compared with 2.7% after the project's implementation. Most patients who needed blood transfusions had not been tested for iron deficiency. While this difference was not statistically significant, Dr. Godby said, it suggests that a reduction in the need for post-partum blood transfusions could be an additional benefit of treating iron deficiency during pregnancy. Dr. Godby noted that nearly all of the patients in both the Before and After cohorts took prenatal vitamins, which are recommended during pregnancy and supposed to contain iron. However, these supplements were usually purchased over the counter rather than prescribed by the health care team. Over-the-counter dietary supplements are not regulated to ensure they contain the ingredients and amounts of ingredients claimed by the manufacturers, he said. As a next step, the team hopes to analyze whether treating iron deficiency in pregnancy improves patients' quality of life by enabling them to feel better, experience less post-partum depression, return to work sooner, and more. Richard Godby, MD, of the Mayo Clinic, will present this study on Sunday, December 7, 2025, at 12:00 noon Eastern time in W304A-D of the Orange County Convention Center. IV Iron Improves Survival, Increases Hemoglobin in Hospitalized Patients with Iron-Deficiency Anemia and an Acute Infection 5: Deciphering the dilemma: Intravenous (IV) iron use in iron deficiency anemia during acute infections Treatment with intravenous (IV) iron significantly improved survival and increased hemoglobin levels in patients with iron-deficiency anemia who were hospitalized for an acute bacterial infection, according to an analysis of data from more than 85,000 patients. "Our data show that it is safe to give IV iron to patients who have both iron-deficiency anemia and an acute bacterial infection, and that, compared with untreated patients, those treated with IV iron have better overall survival and higher hemoglobin levels," said lead author Haris Sohail, MD, a fellow in hematology-oncology at Charleston Area Medical Center in West Virginia. IV iron is a standard treatment for severe iron-deficiency anemia, but its use in patients with both iron-deficiency anemia and an acute bacterial infection is controversial, Dr. Sohail said. Studies have shown that in laboratory experiments, certain bacteria can multiply when iron is added, he said. Although this effect has not been confirmed in human studies, he said, treatment guidelines have long recommended against giving IV iron to patients with active bacterial infections due to concern that the treatment could worsen the infection. In this study, Dr. Sohail and his colleagues searched a large database of de-identified data from patients treated at medical centers across the United States for patients aged 18 and over with iron-deficiency anemia who were hospitalized with an acute bacterial infection between 2000 and 2024. They collected data for over 85,000 patients with the five most common acute bacterial infections treated in U.S. hospitals – over 27,000 with pneumonia, over 23,000 with urinary tract infections, over 15,000 with methicillin-resistant staphylococcus aureus (MRSA), over 13,000 with cellulitis (a skin infection), and over 7,000 with colitis (inflammation of the colon) – as well as 143 patients with bacterial meningitis (inflammation of the membranes covering the brain and spinal cord). The researchers then compared outcomes for patients who received IV iron treatment and those who did not. They looked at how many patients in each group died within 14 or 90 days, how long they spent in the hospital, and how much their hemoglobin levels increased at 60 to 90 days after IV iron treatment compared with the levels before treatment. For every infection except meningitis, results showed that patients treated with IV iron were statistically significantly less likely to die within 14 or 90 days and had larger increases in hemoglobin levels than patients who did not receive IV iron. In patients with meningitis, IV iron did not improve survival, but did not worsen outcomes. "The survival benefit of IV iron was seen across different types of infections, with the biggest improvements seen in patients with pneumonia, MRSA bacteria in the blood, and colitis," Dr. Sohail said. While patients who received IV iron stayed slightly longer in the hospital, the difference was small – only about four to six hours – and not considered clinically meaningful, he said. The small number of patients with bacterial meningitis likely explains why the findings for that infection were not statistically significant, Dr. Sohail said. He added that because the study reviewed the records of patients treated in the past, it can show only an association between IV iron and patient outcomes but cannot prove causation. Additional study limitations, he said, are that the database the researchers used did not provide detailed information about specific bacteria or iron doses, and the findings mainly apply to hospitalized patients with both iron-deficiency anemia and an active bacterial infection. "Our findings support consideration of the use of IV iron as a safe additional therapy for patients who are hospitalized with both iron-deficiency anemia and an acute bacterial infection," he said, adding that a next step would be to confirm these findings in a randomized controlled trial. Haris Sohail, MD, of Charleston Area Medical Center, will present this study on Sunday, December 7, 2025, at 3:25 p.m. Eastern time during the plenary scientific session in West Hall D2 of the Orange County Convention Center. Black Patients with Acute Myeloid Leukemia are Younger at Diagnosis and Experience Poorer Survival Outcomes Than White Patients 290: Inferior survival in black AML patients treated with intensive chemotherapy in ECOG-ACRIN clinical trials is independent of cytogenetic profiles Compared with white patients, Black patients with acute myeloid leukemia (AML) were on average more than five years younger at diagnosis, more than 30% more likely to die of their disease, and more than 20% more likely to die of any cause, according to an analysis of data conducted over a 34-year period and supported by the National Cancer Institute (NCI) is a component of the U.S. National Institutes of Health. Among patients with a mutation in their cancer cells that is generally associated with more favorable outcomes from AML treatment, survival for Black patients was less than half that of white patients. "To our knowledge, this study includes data for a larger number of Black patients than any other such study of AML survival across NCI-supported clinical trials," said study first author Shella Saint Fleur-Lominy, MD, PhD, an associate professor of medicine at the University of Maryland School of Medicine and a member of the cancer therapeutics program at the University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center. "Our findings confirm those of previous, smaller studies that Black patients with AML develop the disease at a significantly younger age, on average, than white patients and, even when treated in clinical trials, have significantly worse outcomes than white patients." AML is a rare, fast-growing cancer that starts in the bone marrow, the soft tissue inside bones where blood cells form. Early blood-forming cells change (mutate) and begin to grow out of control, resulting in large numbers of abnormal, immature blood cells that spill into the bloodstream and crowd out healthy cells. As a result, AML can cause organs to stop working properly and make the body very sick. Doctors base treatment recommendations on biopsy tests that identify the "profile" of mutations in a patient's AML cells because different drugs can target different mutations, leading to better responses to treatment and longer survival. Years of published reports have shown poorer outcomes for Black patients treated for AML compared with white patients, Dr. Saint Fleur-Lominy said; the reasons for this disparity are not completely understood. In one recent study, Black patients diagnosed in their teens or under age 40 had worse outcomes than their white counterparts even when they had mutations that, in white patients, are usually associated with more favorable treatment outcomes. Clinical trials often allow patients with cancer to access novel therapies for their disease before they become widely available. After decades of efforts to promote access to clinical trials for patients of all backgrounds, a 2020 report showed an increase in the number of Black patients in trials supported by the NCI. A related study found a higher rate of Black patients in NCI-sponsored trials than in pharmaceutical company trials. Still, Black patients remain underrepresented in many clinical trials, Dr. Saint Fleur-Lominy said. In this study, she and her colleagues reviewed the medical records of all patients with newly diagnosed AML who were enrolled in 10 ECOG-ACRIN Cancer Research Group clinical trials between 1984 and 2019 (ECOG-ACRIN is a member of the NCI's National Clinical Trials Network). The research team then compared white and Black patients' overall survival (i.e., death from any cause), disease-free survival (i.e., the length of time after treatment during which a patient has no signs of the disease returning), and the genetic mutations found in their cancer cells. They collected data for a total of 3,469 white patients, 184 Black patients, and 156 patients of other races and ethnicities. Across the 10 trials, the proportion of Black patients enrolled ranged from around 1% to 11%. The median age of Black patients at AML diagnosis was 47.9 years, compared with 53.5 years for white patients, a statistically significant difference. Moreover, compared with white patients, Black patients had a 31.3% higher risk of dying from AML and 21.2% higher risk of dying from any cause. Results showed that the most common mutations seen in AML cells occurred at similar rates in Black and white patients. However, among patients with a mutation in one particular gene, known as NPM1, the analysis found a significant difference in overall survival, with Black patients surviving for a median of 8.9 months compared with 19.1 months for white patients. "Although the NPM1 mutation is typically associated with more favorable outcomes of AML treatment, we did not see those more favorable outcomes in Black patients," said Dr. Saint Fleur-Lominy. While similar numbers of Black and white patients overall received transplants of blood-forming stem cells, fewer Black patients (37.1%) than white patients (48.5%) received stem cells from a healthy, compatible donor. This type of transplant offers patients with high-risk AML the best chance of a cure, Dr. Saint Fleur-Lominy said. The alternative, collecting the patient's own stem cells and reinfusing them after high-dose chemotherapy, is no longer considered a standard treatment for AML in the United States, she added. A limitation of the study, she said, is that data on patients' mutations profiles are less comprehensive than she and her team would have liked. The study includes data for patients treated across four decades, she said, and the advanced technology now available to perform such testing did not exist when clinical trials of AML treatment were conducted in earlier decades. As a next step, Dr. Saint Fleur-Lominy said that she would like to see the data from this study combined with other data sets of patients with AML to increase the number of patients with comprehensive genetic data and confirm whether the association of certain genetic mutations with AML outcomes differs in Black patients compared with white patients. This study was conducted by the ECOG-ACRIN Cancer Research Group and supported by the NCI. Shella Saint Fleur-Lominy, MD, PhD, of the University of Maryland School of Medicine, will present this study on Saturday, December 6, 2025, at 2:15 p.m. Eastern time in W415A (Valencia Room) of the Orange County Convention Center. Roughly One-Third of Families with Children Being Treated for Leukemia Struggle to Pay Living Expenses 710: Cumulative incidence of household material hardship and income loss as measures of financial toxicity during pediatric acute lymphoblastic leukemia (ALL) treatment: A report from the DFCI ALL 16-001 Trial Nearly a third of families with children receiving chemotherapy for acute lymphoblastic leukemia (ALL) – the most common pediatric cancer – develop serious financial difficulties during their child's treatment, including losing 25% or more of their household income and struggling to cover the costs of basic living expenses such as housing, food, and utilities. "To our knowledge, this is the first study in pediatric oncology to examine the financial impact associated with cancer treatment by measuring household material hardship and income loss over time," said first author Daniel Zheng, MD, an attending oncologist with the Cancer Center at Children's Hospital of Philadelphia, and faculty member at PolicyLab at Children's Hospital of Philadelphia. "Many families reported experiencing financial difficulties as early as six months into treatment and nearly one in four families who reported no financial hardship at the time their child was diagnosed with cancer developed hardship during the child's treatment." Around 3,000 new cases of ALL are diagnosed each year in the United States. While more than 90% of children treated for ALL survive, Dr. Zheng said, successful treatment typically involves at least two years of multidrug chemotherapy. "That's a lot of clinic visits, hospitalizations, and disruption of a family's day-to-day life." Having a child with cancer places a special burden on families, he said. For example, a caregiver may have to take time off work to take the child to their appointments or stay with them during hospitalizations. "We wanted to try to understand the nature and extent of the financial hardship that treatment for childhood cancer creates for families so that we can better support them during this journey," he said. The study involved children enrolled in a clinical trial of therapies for previously untreated ALL between 2017 and 2021 at eight medical centers in the U.S. and Canada. Participants under age 18 and their families were invited to also take part in an analysis of the financial impact of ALL treatment. The study's primary endpoint was "financial toxicity," defined as the development of new financial hardship (i.e. inability to pay for housing, food, or utilities, or the loss of at least 25% of household income) during the two years of their child's chemotherapy treatment. A total of 422 families (88% of those eligible) opted to participate. Of these families, 15% were Hispanic and 7% non-Hispanic Black; 23% were single-parent households; and 40% had an annual income below 200% of the federal poverty level. They completed questionnaires four times (within about 30 days of diagnosis and at six, 12, and 24 months), which asked the families their household income and whether they were able to cover the costs of housing, food, or utilities at each time point. At six months, 19.3% of families said they had experienced new difficulties covering the cost of housing, food, or utilities, and 20.3% said they had lost 25% or more of their annual income since the start of treatment. By the end of chemotherapy at 24 months, 30% of families had experienced difficulty covering living expenses and 31.5% had lost 25% or more of their income during treatment. Notably, among the 307 families who started treatment reporting no financial hardships, 24.3% experienced new difficulties meeting basic living costs and 27.9% reported losing 25% or more of their income during treatment. Children identified as Hispanic or non-Hispanic Black, who spoke a language other than English at home, lived in single-parent households, had public insurance (e.g. Medicaid), or lived in households with an income below 200% of the federal poverty line at diagnosis were more likely to develop new financial toxicity during the study period. "It was striking to us that by 24 months, nearly a third of the families were unable to meet basic living costs at some point during their child's ALL treatment," Dr. Zheng said. A limitation of the study is that the findings are restricted to patients who were participating in a clinical trial at centers in the northeastern U.S. and Canada, and who were able to successfully complete the surveys at multiple time points. "Our data may underestimate the overall level of financial toxicity experienced by families with children being treated for ALL," Dr. Zheng said. Research is ongoing to identify interventions to address financial toxicity among families of children receiving cancer treatment, he said. For example, two co-authors on this study (Puja Umaretiya, MD, and Kira Bona, MD, MPH) are leading clinical trials examining whether benefits counseling and cash payments reduce financial hardship among low-income families whose children are receiving cancer treatment. Daniel Zheng, MD, of Children's Hospital of Philadelphia, will present this study on Sunday, December 7, 2025, at 4:45 p.m. Eastern time in W230 of the Orange County Convention Center. The American Society of Hematology (ASH) (hematology.org) is the world's largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. Since 1958, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. Join the #Fight4Hematology by visiting hematology.org/fight4hematology. The Blood journals () are the premier source for basic, translational, and clinical hematologic research. The Blood journals publish more peer-reviewed hematology research than any other academic journals worldwide. SOURCE American Society of Hematology 21% more press release views with Request a Demo

369 patients enrolled and 115 treated since FDA approval of ZYNTEGLO ™ and LYFGENIA ™ SOMERVILLE, Mass.--(BUSINESS WIRE)--Genetix Biotherapeutics Inc. today announced their participation in the American Society of Hematology’s (ASH) exclusive press program ahead of the 67th ASH annual meeting in Orlando, Florida (December 6-9, 2025). Genetix’s abstract Accelerating access to gene therapy: Lessons from commercial implementation in sickle cell disease and transfusion-dependent thalassemia was selected by the ASH Program Committee as one of the meeting’s most impactful datasets submitted this year. “We’re honored to be selected for ASH’s exclusive program, a recognition that underscores the significance of our data, which is the first to analyze commercial gene therapy adoption for the treatment of hemoglobinopathies,” said Joanne Lager, MD, Chief Medical Officer. “These findings from patients prescribed ZYNTEGLO and LYFGENIA demonstrate the growing demand and scalability of our durable one-time therapies, setting a new benchmark for delivering commercial gene therapy to patients seeking curative therapies. Building on lessons learned from our first launch of ZYNTEGLO, we’ve accelerated access to LYFGENIA for sickle cell patients, shortening timelines and expanding treatment availability. While initial progress has been made, the vast majority of the thousands of individuals who would benefit from receiving LYFGENIA have not yet been treated. Genetix remains determined and committed to invest in its commercial infrastructure throughout the U.S. to further expand access to LYFGENIA to those in need.” Since FDA approval of ZYNTEGLO (August, 2022) and LYFGENIA (December, 2023), nearly 370 patients have initiated the treatment journey of which 115 have been treated as of mid-November. Lessons learned from the ZYNTEGLO launch along with Genetix’s revamped commercial strategy and ongoing expansion of infrastructure is enabling faster, broader access to LYFGENIA for patients with sickle cell disease. LYFGENIA Commercial Launch Metrics Compared to ZYNTEGLO 2x faster lead time from FDA approval to first patient enrollment 167% faster enrollment rates in the first quarter post approval 6x more treatment center activations in the first quarter post approval Genetix will present the full dataset in an oral presentation at the annual ASH meeting. See details below. Abstract Title: Accelerating Access to Gene Therapy: Lessons from Commercial Implementation in Sickle Cell Disease and Transfusion-Dependent Thalassemia (Abstract #948) Date & Time: Monday, December 8th at 4:00pm EST Please refer to the ASH 2025 online program for full session details and visit the Genetix booth (#1261) onsite. About LYFGENIA™ (lovotibeglogene autotemcel) LYFGENIA is a one-time ex-vivo lentiviral vector (LVV) gene addition therapy approved for eligible patients with sickle cell disease, in which a functional β-globin gene is added to patients’ own hematopoietic (blood) stem and progenitor cells (HSPCs). This addition results in the production of adult hemoglobin with anti-sickling properties (HbAT87Q) which has a similar oxygen-binding affinity to wild-type HbA. LYFGENIA has been shown to limit sickling of red blood cells and reduce or eliminate vaso-occlusive events (VOEs). LYFGENIA is indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of VOEs. Limitations of Use Following treatment with LYFGENIA, patients with α-thalassemia trait (-α3.7/-α3.7) may experience anemia with erythroid dysplasia that may require chronic red blood cell transfusions. LYFGENIA has not been studied in patients with more than two α-globin gene deletions. IMPORTANT SAFETY INFORMATION FOR LYFGENIA (lovotibeglogene autotemcel) Boxed WARNING: HEMATOLOGIC MALIGNANCY Hematologic malignancy has occurred in patients treated with LYFGENIA. Monitor patients closely for evidence of malignancy through complete blood counts at least every 6 months and through integration site analysis at Months 6, 12, and as warranted. Hematologic Malignancy Hematologic malignancy has occurred in patients treated with LYFGENIA (Study 1, Group A). At the time of initial product approval, two patients treated with an earlier version of LYFGENIA using a different manufacturing process and transplant procedure (Study 1, Group A) developed acute myeloid leukemia (AML). One patient with α-thalassemia trait (Study 1, Group C) has been diagnosed with myelodysplastic syndrome (MDS). The additional hematopoietic stress associated with mobilization, conditioning, and infusion of LYFGENIA, including the need to regenerate the hematopoietic system, may increase the risk of a hematologic malignancy. Patients with sickle cell disease have an increased risk of hematologic malignancy as compared to the general population. Patients treated with LYFGENIA may develop hematologic malignancies and should have lifelong monitoring. Monitor for hematologic malignancies with a complete blood count (with differential) at least every 6 months for at least 15 years after treatment with LYFGENIA, and integration site analysis at Months 6, 12, and as warranted. In the event that a malignancy occurs, contact Genetix Biotherapeutics at 1-833-999-6378 for reporting and to obtain instructions on collection of samples for testing. Post-Marketing Long Term Follow-Up Study: Patients who intend to receive treatment with LYFGENIA are encouraged to enroll in the study, as available, to assess the long-term safety of LYFGENIA and the risk of malignancies occurring after treatment with LYFGENIA by calling Genetix Biotherapeutics at 1-833-999-6378. The study includes monitoring (at pre-specified intervals) for clonal expansion. Delayed Platelet Engraftment Delayed platelet engraftment has been observed with LYFGENIA. Bleeding risk is increased prior to platelet engraftment and may continue after engraftment in patients with prolonged thrombocytopenia. Two patients (4%) required more than 100 days post treatment with LYFGENIA to achieve platelet engraftment. Patients should be made aware of the risk of bleeding until platelet recovery has been achieved. Monitor patients for thrombocytopenia and bleeding according to standard guidelines. Conduct frequent platelet counts until platelet engraftment and platelet recovery are achieved. Perform blood cell count determination and other appropriate testing whenever clinical symptoms suggestive of bleeding arise. Neutrophil Engraftment Failure There is a potential risk of neutrophil engraftment failure after treatment with LYFGENIA. Neutrophil engraftment failure is defined as failure to achieve three consecutive absolute neutrophil counts (ANC) ≥ 0.5 × 109 cells/L obtained on different days by Day 43 after infusion of LYFGENIA. Monitor neutrophil counts until engraftment has been achieved. If neutrophil engraftment failure occurs in a patient treated with LYFGENIA, provide rescue treatment with the back-up collection of CD34+ cells. Insertional Oncogenesis There is a potential risk of lentiviral vector-mediated insertional oncogenesis after treatment with LYFGENIA. Hypersensitivity Reactions Allergic reactions may occur with the infusion of LYFGENIA. The dimethyl sulfoxide (DMSO) or dextran 40 in LYFGENIA may cause hypersensitivity reactions, including anaphylaxis. Anti-retroviral Use Patients should not take prophylactic HIV anti-retroviral medications for at least one month prior to mobilization and until all cycles of apheresis are completed. There are some long-acting anti-retroviral medications that may require a longer duration of discontinuation for elimination of the medication. If a patient is taking anti-retrovirals for HIV prophylaxis, confirm a negative test for HIV before beginning mobilization and apheresis of CD34+ cells. Hydroxyurea Use Patients should not take hydroxyurea for at least 2 months prior to mobilization and until all cycles of apheresis are completed. If hydroxyurea is administered between mobilization and conditioning, discontinue 2 days prior to initiation of conditioning. Iron Chelation Drug-drug interactions between iron chelators and the mobilization process and myeloablative conditioning agent must be considered. Iron chelators should be discontinued at least 7 days prior to initiation of mobilization or conditioning. Do not administer myelosuppressive iron chelators (e.g., deferiprone) for 6 months post-treatment with LYFGENIA. Non-myelosuppressive iron chelation should be restarted no sooner than 3 months after LYFGENIA infusion. Phlebotomy can be used in lieu of iron chelation, when appropriate. Interference with PCR-based Testing Patients who have received LYFGENIA are likely to test positive by polymerase chain reaction (PCR) assays for HIV due to integrated BB305 LVV proviral DNA, resulting in a possible false-positive PCR assay test result for HIV. Therefore, patients who have received LYFGENIA should not be screened for HIV infection using a PCR-based assay. Adverse Reactions The most common adverse reactions ≥ Grade 3 (incidence ≥ 20%) were stomatitis, thrombocytopenia, neutropenia, febrile neutropenia, anemia, and leukopenia. Three patients died during LYFGENIA clinical trials; one from sudden cardiac death due to underlying disease and two from acute myeloid leukemia who were treated with an earlier version of LYFGENIA using a different manufacturing process and transplant procedure (Study 1, Group A). Pregnancy/Lactation Advise patients of the risks associated with myeloablative conditioning agents, including on pregnancy and fertility. LYFGENIA should not be administered to women who are pregnant, and pregnancy after LYFGENIA infusion should be discussed with the treating physician. LYFGENIA is not recommended for women who are breastfeeding, and breastfeeding after LYFGENIA infusion should be discussed with the treating physician. Females and Males of Reproductive Potential A negative serum pregnancy test must be confirmed prior to the start of mobilization and re-confirmed prior to conditioning procedures and before LYFGENIA administration. Women of childbearing potential and men capable of fathering a child should use an effective method of contraception (intra-uterine device or combination of hormonal and barrier contraception) from start of mobilization through at least 6 months after administration of LYFGENIA. Advise patients of the options for fertility preservation. Please see full Prescribing Information, including Boxed WARNING and Medication Guide for LYFGENIA. About ZYNTEGLO™ (betibeglogene autotemcel) ZYNTEGLO is a first-in-class, one-time ex-vivo LVV gene addition therapy approved for eligible patients with transfusion-dependent beta-thalassemia who require regular red blood cell (RBC) transfusions, in which functional copies of a modified form of the beta-globin gene (βA-T87Q-globin gene) is added into a patient’s own HSPCs. This addition results in the production of adult hemoglobin (HbAT87Q) enabling total hemoglobin to reach normal or near normal levels. ZYNTEGLO has been shown to eliminate the need for regular RBC transfusions. ZYNTEGLO is indicated for the treatment of adult and pediatric patients with beta-thalassemia who require regular RBC transfusions. IMPORTANT SAFETY INFORMATION FOR ZYNTEGLO (betibeglogene autotemcel) Delayed Platelet Engraftment Delayed platelet engraftment has been observed with ZYNTEGLO treatment. Bleeding risk is increased prior to platelet engraftment and may continue after engraftment in patients with prolonged thrombocytopenia; 15% of patients had ≥ Grade 3 decreased platelets on or after Day 100. Patients should be made aware of the risk of bleeding until platelet recovery has been achieved. Monitor patients for thrombocytopenia and bleeding according to standard guidelines. Conduct frequent platelet counts until platelet engraftment and platelet recovery are achieved. Perform blood cell count determination and other appropriate testing whenever clinical symptoms suggestive of bleeding arise. Risk of Neutrophil Engraftment Failure There is a potential risk of neutrophil engraftment failure after treatment with ZYNTEGLO. Neutrophil engraftment failure is defined as failure to achieve three consecutive absolute neutrophil counts (ANC) ≥ 500 cells/microliter obtained on different days by Day 43 after infusion of ZYNTEGLO. Monitor neutrophil counts until engraftment has been achieved. If neutrophil engraftment failure occurs in a patient treated with ZYNTEGLO, provide rescue treatment with the back-up collection of CD34+ cells. Risk of Insertional Oncogenesis There is a potential risk of lentiviral vector (LVV)-mediated insertional oncogenesis after treatment with ZYNTEGLO. Patients treated with ZYNTEGLO may develop hematologic malignancies and should be monitored lifelong. Monitor for hematologic malignancies with a complete blood count (with differential) at Month 6 and Month 12 and then at least annually for at least 15 years after treatment with ZYNTEGLO, and integration site analysis at Months 6, 12, and as warranted. In the event that a malignancy occurs, contact Genetix Biotherapeutics at 1 833-999-6378 for reporting and to obtain instructions on collection of samples for testing. Hypersensitivity Reactions Allergic reactions may occur with the infusion of ZYNTEGLO. The dimethyl sulfoxide (DMSO) in ZYNTEGLO may cause hypersensitivity reactions, including anaphylaxis. Anti-retroviral and Hydroxyurea Use Patients should not take prophylactic HIV anti-retroviral medications or hydroxyurea for at least one month prior to mobilization, or for the expected duration for elimination of the medications, and until all cycles of apheresis are completed. If a patient requires anti-retrovirals for HIV prophylaxis, then confirm a negative test for HIV before beginning mobilization and apheresis of CD34+ cells. Interference with Serology Testing Patients who have received ZYNTEGLO are likely to test positive by polymerase chain reaction (PCR) assays for HIV due to integrated BB305 LVV proviral DNA, resulting in a false-positive test for HIV. Therefore, patients who have received ZYNTEGLO should not be screened for HIV infection using a PCR-based assay. Adverse Reactions The most common non-laboratory adverse reactions (≥20%) were mucositis, febrile neutropenia, vomiting, pyrexia, alopecia, epistaxis, abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder, and pruritus. The most common Grade 3 or 4 laboratory abnormalities (>50%) include neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia. Drug Interactions Drug-drug interactions between iron chelators and the myeloablative conditioning agent must be considered. Iron chelators should be discontinued at least 7 days prior to initiation of conditioning. The prescribing information for the iron chelator(s) and the myeloablative conditioning agent should be consulted for the recommendations regarding co-administration with CYP3A substrates. Some iron chelators are myelosuppressive. After ZYNTEGLO infusion, avoid use of these iron chelators for 6 months. If iron chelation is needed, consider administration of non-myelosuppressive iron chelators. Phlebotomy can be used in lieu of iron chelation, when appropriate. Pregnancy/Lactation Advise patients of the risks associated with conditioning agents, including on pregnancy and fertility. ZYNTEGLO should not be administered to women who are pregnant, and pregnancy after ZYNTEGLO infusion should be discussed with the treating physician. ZYNTEGLO is not recommended for women who are breastfeeding, and breastfeeding after ZYNTEGLO infusion should be discussed with the treating physician. Females and Males of Reproductive Potential A negative serum pregnancy test must be confirmed prior to the start of mobilization and re-confirmed prior to conditioning procedures and before ZYNTEGLO administration. Women of childbearing potential and men capable of fathering a child should use an effective method of contraception (intra uterine device or combination of hormonal and barrier contraception) from start of mobilization through at least 6 months after administration of ZYNTEGLO. Advise patients of the option to cryopreserve semen or ova before treatment if appropriate. Please see full Prescribing Information and Patient Information for ZYNTEGLO. About Genetix Biotherapeutics Genetix Biotherapeutics Inc. is a privately-held, commercial-stage biotechnology company dedicated to delivering genetic therapies for patients with severe rare diseases. Backed by more than 30 years of pioneering genetic therapy innovation, the company has FDA-approved treatments for sickle cell disease, β-thalassemia, and cerebral adrenoleukodystrophy that directly treat the underlying cause of disease. Genetix is committed to commercial execution, scaling patient access, and improving the treatment experience for patients and providers. Genetix is headquartered in Somerville, Massachusetts.