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Medscape
5 days ago
- Health
- Medscape
GVHD in Children: What Dermatologists Need to Know
SEATTLE — With increasing survival rates after allogeneic hematopoietic stem cell transplantation (HSCT) in children, dermatologists may be seeing more skin conditions associated with HSCT, including the cutaneous manifestations of graft-vs-host disease (GVHD), according to Jennifer T. Huang, MD, associate professor of dermatology and pediatrics, Harvard Medical School, Boston. GVHD is the most common complication following HSCT, and while it can affect any body organ, the skin is the most commonly involved. 'In the US, there are 85 centers in 37 states that perform pediatric allogeneic transplants,' Huang said at the Society for Pediatric Dermatology (SPD) 2025 Annual Meeting. In the US, about 1400 allogeneic transplants are performed each year, and 15% are in children, she noted. While HSCT is generally associated with cancer treatment, in about one third of cases in children, allogeneic HSCT is used to treat nonmalignant conditions such as bone marrow failure, primary immunodeficiency, and hemoglobinopathy. Currently, 5-year survival after pediatric allogeneic HSCT ranges from 50% to 90%, said Huang, who is also director of pediatric oncodermatology at Boston Children's Hospital, Boston. As a result, dermatologists may be seeing more cases of not only cutaneous manifestations of GVHD but also other HSCT-associated effects, such as persistent hair loss and skin cancer. The grading criteria for acute GVHD are well established for the skin, liver, and intestinal tract effects. For the skin, severity is staged based on the percentage of body surface area affected by a rash and the presence or absence of blistering. Generalized erythroderma and the presence of blisters indicate a more severe stage of GVHD. When it presents with symptoms such as generalized erythroderma, desquamation, and bullae, acute, severe GVHD can mimic toxic epidermal necrolysis (TEN), a severe drug-induced skin reaction characterized by widespread epidermal necrosis. Because transplant recipients often take many medications, it can be difficult to distinguish between the two conditions. TEN-like GVHD is rare, noted Huang, a co-author of a case series and literature review of TEN-like acute GVHD in pediatric bone marrow transplant patients. 'Of the 10 patients in the series with TEN-like GVHD, seven had other organ involvement, and 80% died,' she said. Managing Acute GVHD When managing a patient with acute GVHD with cutaneous manifestations, a biopsy may not be very helpful. She referred to a small study conducted at Boston Children's Hospital of 27 children who underwent a biopsy for skin eruptions suspicious for acute GVHD. The biopsy yielded a definitive diagnosis of GVHD or other disorder in only four (15%) cases and changed management in only two patients. In contrast, a dermatology consultation was associated with changes in clinical management in nearly 80% of the patients (21 of the 27) compared with 7.4% (2 of the 27) of skin biopsy results. 'We should be careful about doing biopsies in these patients, unless you think that there's another diagnosis that's a slam dunk histopathological diagnosis like leukemia cutis,' she said. 'Otherwise, you cannot differentiate GVHD from drug rash or viral exanthem, and you should rely on your clinical signs and symptoms.' Second, dermatologists need to know the details of the transplant to evaluate the risk, and third, 'look for acral involvement and systemic symptoms to lead you to the diagnosis, and don't rely on skin biopsy,' she said. 'Importantly, treat aggressively and early, especially if there is TEN-like GVHD.' Chronic GVHD Acute GVHD is the most important risk factor for developing chronic GVHD, a complex, multisystem disorder that is distinct from acute GVHD, with features that resemble autoimmune and other immunologic conditions. 'I think of chronic GVHD as alloimmunity,' Huang explained. Continuing inflammation activates an 'alloresponse,' resulting in the development of what appears to be an autoimmune disease affecting the skin and other organs 'as a response to chronic inflammation,' she said. Chronic skin GVHD most commonly presents in children with eczematous, sclerotic, or lichenoid skin changes and can often be diagnosed clinically without a biopsy in many cases. If a patient presents with diagnostic features such as poikiloderma, lichen planus-like features, sclerotic features, morphea-like features, and lichen sclerosus-like features, biopsy confirmation is generally not required, she noted. Regardless of whether it's acute or chronic, there are minimal histopathologic criteria for diagnosing active GVHD, Huang said. These include the presence of apoptosis in the epidermal basal layer or lower Malpighian layer or outer root sheath of hair follicles, or acrosyringium, lichenoid inflammation, vacuolar changes, and lymphocytic satellitosis. Management of chronic GVHD is usually multifactorial, with three main goals: to improve quality of life (addressing symptoms, daily activities, and visible changes); to minimize systemic immunosuppression; and to avoid permanent disfigurement. 'There are effective topical therapies for skin GVHD that can minimize systemic immunosuppression, and these can often do the trick,' which include steroids, as well as tacrolimus and ruxolitinib, she said. Preventive measures are underutilized, such as gentle care for dry skin, treating other rashes, and, very importantly, sun protection, because sunburn can trigger GVHD, she added. Phototherapy is also a good therapeutic option for skin-directed therapy. In addition, there are now several FDA-approved targeted therapies for chronic GVHD in children, including ibrutinib, ruxolitinib, belumosudil, and axatilimab. 'As dermatologists, we can also guide therapy with biologics that we feel comfortable with but that our other colleagues may not know much about,' said Huang. 'Let's maximize skin-directed therapies for treating this disease. We don't want to lose the graft, and we also don't want permanent disfigurement — this has a lot to do with recognizing the disease early and treating it aggressively.' Huang reported having no relevant financial relationships or conflicts of interest.
Qatar Tribune
03-06-2025
- General
- Qatar Tribune
‘New paediatric Hematopoietic Stem Cell Transplant Ward expands access to life-saving care for children'
Dr Iyabo Tinubu-Karch, CEO of Sidra Medicine, said: 'The opening of our new paediatric Hematopoietic Stem Cell Transplant Ward, the first of its kind in the country, marks a major step in expanding access to life-saving care for children in Qatar and the region. As the HSCT program grows, it will play a vital role in reducing the need for outbound referrals and in positioning Qatar as a center of excellence for pediatric cell and gene therapies. This is about building sustainable, high-quality care close to home for our patients and their families.' Prof. Ibrahim Janahi, Chief Medical Officer at Sidra Medicine, said: 'The launch of the HSCT Ward reflects our commitment to supporting the Qatar National Vision 2030. It strengthens the foundation of a knowledge-based economy by building national expertise in precision and regenerative medicine. Our goal is to ensure that every child in Qatar can access the best possible treatment, close to home and grounded in clinical excellence.' Managed by the Hematology and Oncology Division at Sidra Medicine, the new ward is designed exclusively for pediatric patients under the age of 18. It is divided into two main sections—one section for children undergoing HSCT and the other for patients with complex hematology-oncology diagnoses such as cancer, that require intensive chemotherapy and inpatient care. With an initial capacity of 20 beds, the new pediatric HSCT ward integrates cutting-edge infrastructure, including high-level isolation facilities that meet international infection control standards. It is scheduled to receive patients for autologous transplants in late June of 2025 with preparations underway to care for inpatient children who will undergo allogeneic transplants, at a later stage. Dr Ayman Saleh, Division Chief of Pediatric Hematology-Oncology & Hematopoietic Stem Cell Transplant at Sidra Medicine, said: 'Our HSCT program brings together key specialties to deliver advanced and curative care for children with complex conditions. With four autologous procedures successfully completed and plans underway to offer allogeneic transplants in the near future, we are building a strong foundation for pediatric cellular therapy in Qatar. This service is a testament to the pioneering and family-centered care and cure approach that we offer to all our patients.' In addition to treating patients with various blood diseases and cancers, the HSCT program at Sidra Medicine is the core part of its precision medicine strategy to facilitate genomic research programs for pediatric autoimmune disorders, genetic and rare diseases. Sidra Medicine's HSCT program is further enhanced by a Good Manufacturing Practice (GMP)-qualified laboratory team operating under the Advanced Cell Therapy Core (ACTC). The ACTC manages the processing and cryopreservation of hematopoietic stem cells and is currently working toward Sidra Medicine's qualification as a treatment center for Casgevy, a gene therapy for thalassemia and sickle cell disease. Dr Chiara Cugno, Director of the Advanced Cell Therapy Core and GMP Facility at Sidra Medicine said: 'Our GMP-qualified laboratory is a key enabler in delivering safe and effective cellular therapies. By maintaining the highest standards in cell processing and cryopreservation, we are supporting precision treatments for children with cancer, blood, and immunologic disorders. It is an essential part of ensuring every child receives the most personalized care possible.' All HSCT program services will be open to both local and international patients, with more than 50 children currently awaiting transplantation in Qatar. Future phases for the HSCT program include scaling allogeneic transplant capacity and introducing CAR-T cell and gene therapies. The HSCT program includes a multidisciplinary team of specialized physicians, apheresis-trained staff, dedicated HSCT nurses, and allied health professionals. The Sidra Medicine care team also involves experts in immunology, infectious diseases, radiology, psychology, pharmacy, nutrition, child life, and social work. The integrated approach ensures comprehensive and patient-centered care throughout the transplant journey. Before a transplant, every child will go through a detailed evaluation at Sidra Medicine's outpatient clinic to ensure they are ready for the procedure. Once admitted, the children will undergo conditioning chemotherapy to prepare their bodies for the stem cell infusion. They will then be monitored very closely during the aplasia phase, when their immune system is at its weakest. Most patients will stay at the hospital for about 30 to 40 days. Following discharge, they will continue their care through regular follow-ups in an outpatient setting. In addition to medical and nursing care, Sidra Medicine offers support services for patients and families, including social work, clinical nurse specialists, and case management. While formal support groups are under development, ongoing engagement with families is embedded throughout the care process.
Qatar Tribune
03-06-2025
- General
- Qatar Tribune
Sheikha Moza opens HSCT ward at Sidra Medicine
Tribune News Network Doha HH Sheikha Moza bint Nasser, Chairperson of Qatar Foundation for Education, Science and Community Development, officially inaugurated the new paediatric Hematopoietic Stem Cell Transplant (HSCT) ward at Sidra Medicine, a Qatar Foundation entity. The new ward is dedicated to treating children with blood disorders, immune system diseases, and genetic conditions. During the visit, Sheikha Mozatoured the facility, engaged with clinical teams, and received an overview of the HSCT programme's role in advancing care and stem cell and gene therapies for children in Qatar and the region. Sheikha Moza was accompanied by Minister of Public Health HE Mansoor bin Ebrahim bin Saad Al Mahmoud, members of Sidra Medicine's Board of Directors and senior leadership, including Dr Iyabo Tinubu-Karch, Chief Executive Officer and Prof Ibrahim Janahi, Chief Medical Officer. The HSCT ward is a core part of Sidra Medicine's HSCT Programme, using curative therapies to provide life-saving care and treatment for children with a range of blood diseases and cancers. This includes malignant and non-malignant conditions such as leukemia, lymphoma, thalassemia, sickle cell disease, immune deficiencies, and metabolic disorders. HSCT also known as bone marrow transplant, replaces diseased or damaged bone marrow with healthy stem cells from the patient (autologous) or a donor (allogeneic). Dr Iyabo Tinubu-Karch, CEO of Sidra Medicine, said: 'The opening of our new pediatric Hematopoietic Stem Cell Transplant Ward, the first of its kind in the country, marks a major step in expanding access to life-saving care for children in Qatar and the region. As the HSCT program grows, it will play a vital role in reducing the need for outbound referrals and in positioning Qatar as a center of excellence for pediatric cell and gene therapies. This is about building sustainable, high-quality care close to home for our patients and their families.' Prof. Ibrahim Janahi, Chief Medical Officer at Sidra Medicine, said: 'The launch of the HSCT Ward reflects our commitment to supporting the Qatar National Vision 2030. It strengthens the foundation of a knowledge-based economy by building national expertise in precision and regenerative medicine. Our goal is to ensure that every child in Qatar can access the best possible treatment, close to home and grounded in clinical excellence.' Managed by the Hematology and Oncology Division at Sidra Medicine, the new ward is designed exclusively for pediatric patients under the age of 18. It is divided into two main sections - one section for children undergoing HSCT and the other for patients with complex hematology-oncology diagnoses such as cancer, that require intensive chemotherapy and inpatient care. With an initial capacity of 20 beds, the new pediatric HSCT ward integrates cutting-edge infrastructure, including high-level isolation facilities that meet international infection control standards. It is scheduled to receive patients for autologous transplants in late June of 2025 with preparations underway to care for inpatient children who will undergo allogeneic transplants, at a later stage. Dr Ayman Saleh, Division Chief of Pediatric Hematology-Oncology & Hematopoietic Stem Cell Transplant at Sidra Medicine said: 'Our HSCT program brings together key specialties to deliver advanced and curative care for children with complex conditions. With four autologous procedures successfully completed and plans underway to offer allogeneic transplants in the near future, we are building a strong foundation for pediatric cellular therapy in Qatar. This service is a testament to the pioneering and family-centered care and cure approach that we offer to all our patients.' In addition to treating patients with various blood diseases and cancers, the HSCT program at Sidra Medicine is the core part of its precision medicine strategy to facilitate genomic research programs for pediatric autoimmune disorders, genetic and rare diseases. Sidra Medicine's HSCT program is further enhanced by a Good Manufacturing Practice (GMP)-qualified laboratory team operating under the Advanced Cell Therapy Core (ACTC). The ACTC manages the processing and cryopreservation of hematopoietic stem cells and is currently working toward Sidra Medicine's qualification as a treatment center for Casgevy, a gene therapy for thalassemia and sickle cell disease. Dr Chiara Cugno, Director of the Advanced Cell Therapy Core and GMP Facility at Sidra Medicine said: 'Our GMP-qualified laboratory is a key enabler in delivering safe and effective cellular therapies. By maintaining the highest standards in cell processing and cryopreservation, we are supporting precision treatments for children with cancer, blood, and immunologic disorders. It is an essential part of ensuring every child receives the most personalized care possible.' All HSCT program services will be open to both local and international patients, with more than 50 children currently awaiting transplantation in Qatar. Future phases for the HSCT program include scaling allogeneic transplant capacity and introducing CAR-T cell and gene therapies. The HSCT program includes a multidisciplinary team of specialized physicians, apheresis-trained staff, dedicated HSCT nurses, and allied health professionals. The Sidra Medicine care team also involves experts in immunology, infectious diseases, radiology, psychology, pharmacy, nutrition, child life, and social work. The integrated approach ensures comprehensive and patient-centered care throughout the transplant journey. Before a transplant, every child will go through a detailed evaluation at Sidra Medicine's outpatient clinic to ensure they are ready for the procedure. Once admitted, the children will undergo conditioning chemotherapy to prepare their bodies for the stem cell infusion. They will then be monitored very closely during the aplasia phase, when their immune system is at its weakest. Most patients will stay at the hospital for about 30 to 40 days. Following discharge, they will continue their care through regular follow-ups in an outpatient setting. In addition to medical and nursing care, Sidra Medicine offers support services for patients and families, including social work, clinical nurse specialists, and case management. While formal support groups are under development, ongoing engagement with families is embedded throughout the care process.
Qatar Tribune
03-06-2025
- General
- Qatar Tribune
Spotlight on SidraMedicine's Stem Cell Transplant Ward
Tribune News Network Doha The new Paediatric Hematopoietic Stem Cell Transplant (HSCT) Ward at Sidra Medicine represents a major advancement in its commitment to deliver state-of-the-art, lifesaving care for children with cancer, blood disorders, immune deficiencies, and other complex conditions. As part of Sidra Medicine's comprehensive Paediatric Hematology—Oncology, and HSCT Programme, the ward is uniquely equipped to provide curative therapy through cellular and gene-based treatment approaches. This initiative positions Sidra Medicine as a national and regional leader in paediatric stem cell transplantation and personalized medicine. 1. What is the purpose of the new paediatric HSCT Ward at Sidra Medicine? The new paediatric HSCT Ward is designed to treat children with life-threatening cancer, blood, immune, and genetic disorders. It is a core part of Sidra Medicine's HSCT Programme using curative therapy to provide life-saving treatment for children. is HSCT and how does it work? HSCT (Hematopoietic Stem Cell Transplantation), also known as bone marrow transplant, replaces diseased or damaged bone marrow with healthy stem cells from the patient (autologous) or a donor (allogeneic). It is currently the only curative treatment for many congenital and acquired otherwise fatal and acquired fatal paediatric diseases. The procedure is at the forefront of personalised medicine and plays a vital role in the development of advanced cell and gene therapies. 3. Why is the paediatric HSCT ward being opened? To address the growing demand for paediatric transplant services, eliminate the need for families to travel abroad, and enhance our national capability in advanced cellular therapy. conditions will be treated at theHSCT Ward? The HSCT ward will serve paediatric patients with a range of malignant and non-malignant conditions, including leukemia, lymphoma, solid tumours, thalassemia, sickle cell disease, primary immune deficiencies, metabolic diseases, and certain autoimmune and genetic disorders. many beds are in the HSCT Ward? The paediatric hematology-oncology and HSCT Ward has a 20-bed capacity. It is divided into two main sections: One section is dedicated to pediatric patients undergoing HSCT (bone marrow transplant), and the other for patients with complex hemato-oncology diagnoses, such as paediatric cancer, who are receiving chemotherapy or inpatient care for treatment-related complications. Note that HSCT is a medical—not surgical—procedure, and no surgeries take place in the HSCT ward. will the paediatric HSCT ward open? The paediatric HSCT ward is scheduled to receive patients for autologous transplants in late June of 2025 with preparations underway to care for inpatient children who will undergo allogeneic transplants at a later stage. is eligible for treatment? The ward is exclusively for paediatric patients under the age of 18. It serves both local and international patients in need of HSCT or inpatient hematology-oncology care. is the HSCT Programme about? The HSCT programme at Sidra Medicine is part of a precision medicine strategy to treat patients with various blood diseases and cancers including sickle cell disease, thalassemia, leukemia, lymphoma, solid tumors. In addition, the programme includes the treatment of primary immune deficiencies, metabolic diseases, and certain genetic and autoimmune disorders. makes this programme unique in Qatar? This is the first dedicated paediatric HSCT programme in the country, reducing the need for children to travel abroad for transplant services. The programme also integrates cutting-edge research, cell therapy, and gene therapy capabilities. 10. What are the safety and infection control measures in place? The unit adheres to the highest international infection control standards, including high-level HEPA-filtered, positive pressure isolation rooms to protect patients during the vulnerable aplasia period when the immune system is suppressed. 11. How many children are currently awaiting HSCT in Qatar? More than 50 children are currently on the transplant waitlist, highlighting the urgent need for this new facility. Sidra Medicine treat international patients? Yes, our services are open to eligible international paediatric patients and align with our vision to be a regional centre of excellence in paediatric cell and gene therapy.
Qatar Tribune
03-06-2025
- General
- Qatar Tribune
Sheikha Moza inaugurates new paediatric HSCT ward at Sidra Medicine
DOHA: Chairperson of Qatar Foundation for Education, Science and Community Development HH Sheikha Moza bint Nasser has officially inaugurated the new paediatric Hematopoietic Stem Cell Transplant (HSCT) ward at Sidra Medicine, a Qatar Foundation entity. The new ward is dedicated to treating children with blood disorders, immune system diseases, and genetic conditions. During the visit, Sheikha Moza toured the facility, engaged with clinical teams, and received an overview of the HSCT program's role in advancing care and stem cell and gene therapies for children in Qatar and the region. Her Highness was accompanied by Minister of Public Health HE Mansoor bin Ebrahim bin Saad Al Mahmoud, members of Sidra Medicine's Board of Directors and senior leadership, including Dr Iyabo Tinubu-Karch, Chief Executive Officer and Prof. Ibrahim Janahi, Chief Medical Officer. The HSCT ward is a core part of Sidra Medicine's HSCT Program, using curative therapies to provide life-saving care and treatment for children with a range of blood diseases and cancers. This includes malignant and non-malignant conditions such as leukemia, lymphoma, thalassemia, sickle cell disease, immune deficiencies, and metabolic disorders. HSCT also known as bone marrow transplant, replaces diseased or damaged bone marrow with healthy stem cells from the patient (autologous) or a donor (allogeneic). Dr Iyabo Tinubu-Karch, CEO of Sidra Medicine, said: "The opening of our new pediatric Hematopoietic Stem Cell Transplant Ward, the first of its kind in the country, marks a major step in expanding access to life-saving care for children in Qatar and the region. As the HSCT program grows, it will play a vital role in reducing the need for outbound referrals and in positioning Qatar as a center of excellence for pediatric cell and gene therapies. This is about building sustainable, high-quality care close to home for our patients and their families." Prof Ibrahim Janahi, Chief Medical Officer at Sidra Medicine, said: "The launch of the HSCT Ward reflects our commitment to supporting the Qatar National Vision 2030. It strengthens the foundation of a knowledge-based economy by building national expertise in precision and regenerative medicine. Our goal is to ensure that every child in Qatar can access the best possible treatment, close to home and grounded in clinical excellence." Managed by the Hematology and Oncology Division at Sidra Medicine, the new ward is designed exclusively for pediatric patients under the age of 18. It is divided into two main sections - one section for children undergoing HSCT and the other for patients with complex hematology-oncology diagnoses such as cancer, that require intensive chemotherapy and inpatient care. With an initial capacity of 20 beds, the new pediatric HSCT ward integrates cutting-edge infrastructure, including high-level isolation facilities that meet international infection control standards. It is scheduled to receive patients for autologous transplants in late June of 2025 with preparations underway to care for inpatient children who will undergo allogeneic transplants, at a later stage. Dr Ayman Saleh, Division Chief of Pediatric Hematology-Oncology & Hematopoietic Stem Cell Transplant at Sidra Medicine said: "Our HSCT program brings together key specialties to deliver advanced and curative care for children with complex conditions. With four autologous procedures successfully completed and plans underway to offer allogeneic transplants in the near future, we are building a strong foundation for pediatric cellular therapy in Qatar. This service is a testament to the pioneering and family-centered care and cure approach that we offer to all our patients." In addition to treating patients with various blood diseases and cancers, the HSCT program at Sidra Medicine is the core part of its precision medicine strategy to facilitate genomic research programs for pediatric autoimmune disorders, genetic and rare diseases. Sidra Medicine's HSCT program is further enhanced by a Good Manufacturing Practice (GMP)-qualified laboratory team operating under the Advanced Cell Therapy Core (ACTC). The ACTC manages the processing and cryopreservation of hematopoietic stem cells and is currently working toward Sidra Medicine's qualification as a treatment center for Casgevy, a gene therapy for thalassemia and sickle cell disease.
