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NCT Number: NCT06351462

Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients

The long term burden of morbidity and mortality in the natural history of sickle cell disease has not been compared up to date to the risks and mortality of a curative option like bone marrow transplantation in severe sickle-cell disease patients. Given this lack of data, primary-care Sickle Cell Disease (SCD) physicians and transplant physicians are prevented from a factual debate over the benefit/risk ratio for each patient and refining indications of transplant in patients. Therefore, the present study seeks to describe and compare the very long-term outcomes after either Human Leukocyte Antigen (HLA) -matched sibling transplantation (study arm) and "non-transplant care" for severe sickle cell disease SCA patients in order to yield robust comparative data regarding both arms.

The main objective is to assess the benefit of Hematopoietic stem cell transplantation (HSCT) regarding quality of life compared to standard care after 10 years, in patients with severe Sickle Cell Disease (SCD).

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

15 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Study population (exposed-patients), all criteria should be fulfilled:

  • Patients alive with Sickle Cell Anemia (SCA, meaning SS and Sbeta0 sickle cell anemia genotype)
  • Patients transplanted from allogeneic HLA-compatible sibling donor or from sibling cord blood unit from the 1st of January 2000 and the 31st of December 2012, whatever the age at transplant
  • Patients having received conditioning regimen containing busulfan 1mg/kg/dose (or equivalent adjusted body-weight dosage according to recommendation) x 16 doses + cyclophosphamide 200mg/kg total dose + anti-thymoglobuline
  • For patient under 18 years at time of enrolment, signed informed consent from both parental representatives
  • For patient aged 18 years old : signed informed consent
  • Having an affiliation to a social security regime

Control-population (Non-exposed patients) :

For each allografted patient, one non-exposed patient will be matched, based on the following criteria:

  • Gender
  • Age at the date of transplantation of the exposed patient (+/- 1 year)
  • Foetal hemoglobin (HbF) level (+/- 3%) before treatment intensification (defined as the initiation of either hydroxyurea or a transfusion program)
  • Hb level (+/- 0,9 g/dl) before treatment intensification
  • For patient under 18 years at time of enrolment, signed informed consent from both parental representatives
  • For patient above 18 years of age: signed informed consent
  • Having an affiliation of to a social security regime

Exclusion criteria

Study population:

  • Transplantation from donor other than sibling or related cord-blood
  • Conditioning regimen other than busulfan 16mg/kg total dose + cyclophosphamide 200mg/kg total dose + anti-thymoglobuline

For both population:

  • Absence of signed informed consent
  • Having any debilitating medical or psychiatric illness, which preclude understanding of the inform consent as well as optimal treatment and follow-up

Treatment and study plan

Spermogram

Other

Spermogram will be proposed to men

Hospital Anxiety and Depression Scale (HADS)

Other

Anxiety and depression will be evaluated 10 years after HSCT

SF36 Quality of life questionnaire

Other

Quality of life will be evaluated 10 years after HSCT

Psychologist interview

Other

During a follow-up visit

Optional sera banking

Other

One in the study

Optional DNA banking

Other

Once in the study

Primary outcomes

  1. Evaluation of quality of life assessed by SF36

    Time frame: 10 years after HSCT

    It will be assessed by the Short-form 36 (SF36) scale. The Short Form (36) Health Survey is a 36-item measure if health status. The score obtained varies between 0 and 100. The higher the score the less disability.

    Ware JE, Sherbourne CD. The Medical Outcomes Study 36-item short-form health survey (SF-36): I. Conceptual framework and item selection. Med Care 1992;30:473-83.

Secondary outcomes

  1. Evaluation of gonadal function

    Time frame: 10 years after HSCT

    Spermogram in men (proposed not requested), Luteinising Hormone (LH), Follicle-Stimulating Hormone (FSH), estrogen, Anti-Müllerian Hormone (AMH) in women, testosterone in men, Amenorrhea in women.

  2. Evaluation of quality of life

    Time frame: 10 years after HSCT

    Questionnaire including data about employment, education level, social financial support.

  3. Proportion of patients with anxiety and depression

    Time frame: 10 years after HSCT

    Anxiety and depression levels will be assessed using the Hamilton Anxiety Depression scale : The HAD scale is a self-assessment scale for detecting states of depression and anxiety in the setting of an hospital medical outpatient clinic.

    HADS is a self-administered scale of 14 items which assessed levels of depression and anxiety, divided into 2 subscales of 7 items (Anxiety or HADS-A, Depression or HADS-D). Each item is scored on a scale of 0 to 3. A score is generated for each of the two sub-scales (sum of the 7 items, ranging from 0 to 21). Limit scores, for each of the scores, distinguish: non-cases or asymptomatic ones (score ≤ 7); probable or borderline cases (score 8-10); clearly or clinically symptomatic cases (score ≥ 11)

Study contacts

Contact information is provided by the study sponsor or research team.

Jean-Hugues Dalle, Pr

CONTACT

[email protected]

+33140033692

Jérôme Lambert, Pr

CONTACT

[email protected]

+33142499742

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Registry information

Acronym: TRANSPLANTORN2

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Apr 8, 2024
Registry last updated
Apr 19, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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