USAResearch and clinical trials
Intravenous arginine showed no benefit for sickle cell pain crises in a phase 3 trial
The STArT study, published in JAMA, examined arginine in sickle cell pain crises.
Blood and immune system diseases
An inherited blood disease in which red blood cells take on a sickle shape and break down quickly. Anemia, pain attacks and frequent infections may occur.
Not part of the panel of Türkiye's National Newborn Screening Programme (heel-prick test). Source
Orphanet has a guideline for emergency doctors for this disease (language: French). See on Orphanet
This page is translated from the Turkish original.
This disease is followed through the “Blood and immune system diseases” group.
Sickle cell disease is a group of inherited blood diseases. The red blood cells take on a sickle (crescent) shape and become stiff. These cells break down quickly, which causes anemia. They can block small blood vessels and stop oxygen from reaching the tissues.
Symptoms often begin in early childhood. How severe the disease is varies greatly from person to person.
The disease is detected with a blood test. In some health systems, screening is done during pregnancy and in newborns. In newborn screening, a few drops of blood are taken from the baby's heel. Adults can also find out with a blood test whether they are carriers.
The basis of care is relieving pain and preventing infections. For this, preventive medicines and vaccines are used. When needed, a blood transfusion or exchange transfusion is done. For some people, a stem cell transplant or gene therapy may be considered. The doctor who follows the disease decides which treatment is suitable.
To have the disease, a person must receive one changed gene from the mother and one from the father. Carriers often do not have the disease, but the gene can be passed on to the child. If the mother and father are carriers, the risk is 1 in 4 in each pregnancy. Genetic counselling is recommended.
Source: MedlinePlus Genetics — Sickle cell disease · NHS — Sickle cell disease
These facts are taken from Orphanet and MedlinePlus records (last checked: October 7, 2026). Frequency values are approximate ranges.
Non-profit patient and family organizations in Türkiye working on this disease or disease group. Links are for information; Doktorclub is not affiliated with these organizations and does not endorse them.
For all rare diseases: Nadir Hastalıklar Federasyonu
Links checked on October 8, 2026.
Doktorclub does not diagnose, recommend treatments or refer you to hospitals or doctors. These pages are meant to help you talk with your doctor.
USAResearch and clinical trials
The STArT study, published in JAMA, examined arginine in sickle cell pain crises.
USANew treatments and approvals
On July 1, 2026, the US Food and Drug Administration (FDA) approved exagamglogene autotemcel for children over 2 years of age.
A clinical trial is a regulated study that tests whether a new treatment is safe and whether it works, in patients who volunteer. Most trials look for a certain age, genetic change and health condition. Taking part is voluntary.
Questions you can ask your doctor
ClinicalTrials.gov lists 187 active interventional studies for this disease. 111 are recruiting now; 11 have sites in Türkiye. Last checked: October 7, 2026.
Selected records (studies with a site in Türkiye first):
Late stage: the effect is being compared in a large group · Phase 3 · investigational (experimental)RecruitingSite in TürkiyeNCT06609226
Middle stage: the effect is being tested in a small group · Phase 2 · investigational (experimental)RecruitingSite in TürkiyeNCT06198712
Late stage: the effect is being compared in a large group · Phase 3 · investigational (experimental)RecruitingSite in TürkiyeNCT06612268
Middle stage: the effect is being tested in a small group · Phase 1 / Phase 2 · investigational (experimental)RecruitingSite in TürkiyeNCT06546670
Middle stage: the effect is being tested in a small group · Phase 2 · investigational (experimental)RecruitingSite in TürkiyeNCT06699849
See all studies on ClinicalTrials.gov · Joining clinical trials
GlobalHealth policy
The International Pediatric Nephrology Association published its recommendations for diagnosing and treating atypical HUS in children.
GlobalResearch and clinical trials
Researchers re-examined the data from a large drug study.
TürkiyeResearch and clinical trials
A face-to-face survey was conducted with 104 PNH patients at 18 centers in Türkiye.
These codes identify the disease in international classifications and databases. They can help when you talk with your doctor or look for reliable sources.
Orphanet code: ORPHA:275752
No separate ICD-11 code; WHO classifies this disease under: 3A51 (Sickle cell disorders or other haemoglobinopathies)
Codes checked against WHO ICD-11 and Orphanet on October 6, 2026.
This page provides general information and does not replace diagnosis or treatment. Talk to your doctor about symptoms, diagnosis and treatment.