A call to start hydroxyurea by 6 months of age and before the advent of sickle cell disease complications

Elenga, Narcisse; Kayemba-Kay’s, Simon; Archer, Natasha M.

doi:10.1002/pbc.29423

Cited by 3 publications

(5 citation statements)

References 39 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Given substantive improvements in patient symptoms and markers of disease control, the National Heart, Lung, and Blood Institute (NHLBI) recommends offering hydroxyurea in pediatric patients over 9 months of age, regardless of clinical severity ( 68 ). However, the optimal time to start hydroxyurea therapy has not been established, and other national guidelines recommend starting at later ages ( 73 ). Additionally, in patients not taking hydroxyurea, it may be recommended prior to bone marrow transplant to reduce the risk of rejection and improve chance of engraftment ( 74 ).…”

Section: Palliative and Disease Modifying Therapies As Potential Infe...mentioning

confidence: 99%

Fertility preservation for pediatric patients with hemoglobinopathies: Multidisciplinary counseling needed to optimize outcomes

et al. 2022

View full text Add to dashboard Cite

Hemoglobinopathies are autosomal recessive disorders that occur when genetic mutations negatively impact the function of hemoglobin. Common hemoglobinopathies that are clinically significant include sickle cell disease, alpha thalassemia, and beta thalassemia. Advancements in disease-modifying and curative treatments for the common hemoglobinopathies over the past thirty years have led to improvements in patient quality of life and longevity for those who are affected. However, the diseases, their treatments and cures pose infertility risks, making fertility preservation counseling and treatment an important part of the contemporary comprehensive patient care. Sickle cell disease negatively impacts both male and female infertility, primarily by testicular failure and decreased ovarian reserve, respectively. Fertility in both males and females with beta thalassemia major are negatively impacted by iron deposition due to chronic blood transfusions. Hematopoietic stem cell transplant (HSCT) is currently the only curative treatment for SCD and transfusion dependent beta thalassemia. Many of the conditioning regimens for HSCT contain chemotherapeutic agents with known gonadotoxicity and whole-body radiation. Although most clinical studies on toxicity and impact of HSCT on long-term health do not evaluate fertility, gonadal failure is common. Male fertility preservation modalities that exist prior to gonadotoxic treatment include sperm banking for pubertal males and testicular cryopreservation for pre-pubertal boys. For female patients, fertility preservation options include oocyte cryopreservation and ovarian tissue cryopreservation. Oocyte cryopreservation requires controlled ovarian hyperstimulation (COH) with ten to fourteen days of intensive monitoring and medication administration. This is feasible once the patient has undergone menarche. Follicular growth is monitored via transvaginal or transabdominal ultrasound, and hormone levels are monitored through frequent blood work. Oocytes are then harvested via a minimally invasive approach under anesthesia. Complications of COH are more common in patients with hemoglobinopathies. Ovarian hyperstimulation syndrome creates a greater risk to patients with underlying vascular, pulmonary, and renal injury, as they may be less able to tolerate fluids shifts. Thus, it is critical to monitor patients undergoing COH closely with close collaboration between the hematology team and the reproductive endocrinology team. Counseling patients and families about future fertility must take into consideration the patient’s disease, treatment history, and planned treatment, acknowledging current knowledge gaps.

show abstract

Section: Palliative and Disease Modifying Therapies As Potential Infe...mentioning

confidence: 99%

Fertility preservation for pediatric patients with hemoglobinopathies: Multidisciplinary counseling needed to optimize outcomes

et al. 2022

View full text Add to dashboard Cite

show abstract

“…Thus, secondary prevention approaches including improved general SCA care and increased vigilance for clinical suspicion of ICH should be undertaken. Theoretically, strategies to retard EMH using folic acid supplementation and early initiation of hydroxyurea are likely to prevent ASHS 3,17,18 …”

Section: Introductionmentioning

confidence: 99%

“…Theoretically, strategies to retard EMH using folic acid supplementation and early initiation of hydroxyurea are likely to prevent ASHS. 3,17,18 2 | CASE PRESENTATION 2. 1 | Demographic details and medical history LTM was a 16-year-old boy, a primary school student.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Acute soft head syndrome in a teenager with sickle cell anemia: A case report

Magitta,

Komanya,

Alphonce

et al. 2023

Clinical Case Reports

View full text Add to dashboard Cite

Key Clinical MessageSickle cell disease (SCD) rarely presents with acute soft head syndrome (ASHS) often posing a diagnostic dilemma. Recovery is typically spontaneous, however, in the context of lack of awareness and limited brain imaging it could potentially lead to poor outcome.AbstractASHS is a rare complication of SCD, invariably occurring near puberty with hitherto elusive pathogenic mechanisms. ASHS often resolves spontaneously on conservative management, however, lack of awareness in the context of limited access to brain imaging could pose diagnostic challenges resulting in inappropriate management and untoward outcome. We present a case of a teenager who presented with subtle symptoms for which the diagnosis of sickle cell anemia (SCA) was delayed until he developed ASHS. LTM was a 16 years old boy with a history of recurrent joints pain since the age of 6 years, with a family history of SCA, but had initial negative sickling test. He presented with episodes of multiple joints pain, unprovoked scalp and left orbital swelling, low‐grade fever and mild headache without any evidence for bleeding diathesis. The diagnosis of SCA was confirmed by hemoglobin electrophoresis. Computed tomography (CT) scan of the head revealed subgaleal heamatoma (SGH) and intraorbital haematoma without intracranial hemorrhage (ICH). He was managed conservatively with analgesics and hydration together with antibiotics for associated sepsis with complete resolution of clinical symptoms within 2 weeks. This case represents a rare scenario for a relatively mild SCA phenotype presenting with ASHS whose diagnosis poses an enigma in the resource‐limited contex. It is therefore, prudent to recognize ASHS to avoid judicious interventions which could potentially result in untoward clinical outcome.

show abstract

“…In 2014, the Expert Panel Report of the National Heart, Lung, and Blood Institute recommended starting doses of 15 to 20 mg/kg per day for adults and children, with increases of 5 mg/kg per day every 8 weeks “if dose escalation is warranted.” 1 However, escalating the dose to the maximum tolerated dose (MTD) vs maintaining a fixed dose has been controversial, 2 and relatively little has been published regarding hydroxyurea treatment for infants. 3 , 4 , 5 , 6 …”

mentioning

confidence: 99%

Intensive hydroxyurea dosing in very young children with sickle cell anemia

Wang,

Brown,

McNaull

et al. 2023

Blood Advances

View full text Add to dashboard Cite

A call to start hydroxyurea by 6 months of age and before the advent of sickle cell disease complications

Cited by 3 publications

References 39 publications

Fertility preservation for pediatric patients with hemoglobinopathies: Multidisciplinary counseling needed to optimize outcomes

Fertility preservation for pediatric patients with hemoglobinopathies: Multidisciplinary counseling needed to optimize outcomes

Acute soft head syndrome in a teenager with sickle cell anemia: A case report

Intensive hydroxyurea dosing in very young children with sickle cell anemia

Contact Info

Product

Resources

About