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Not yet recruiting 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

No phase Interventional Sickle Cell Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Spermogram, Hospital Anxiety and Depression Scale (HADS), SF36 Quality of life questionnaire, Psychologist interview.
Who it may be relevant to
Registry conditions: Sickle Cell Disease. Basic parameters: 15 years — 80 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Overview

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).

Interventions

  • Other Spermogram
    Spermogram will be proposed to men
  • Other Hospital Anxiety and Depression Scale (HADS)
    Anxiety and depression will be evaluated 10 years after HSCT
  • Other SF36 Quality of life questionnaire
    Quality of life will be evaluated 10 years after HSCT
  • Other Psychologist interview
    During a follow-up visit
  • Other Optional sera banking
    One in the study
  • Other Optional DNA banking
    Once in the study

Primary outcome measures

  • Evaluation of quality of life assessed by SF36 [Time frame: 10 years after HSCT]
Secondary outcome measures (3)
  • Evaluation of gonadal function [Time frame: 10 years after HSCT]
  • Evaluation of quality of life [Time frame: 10 years after HSCT]
  • Proportion of patients with anxiety and depression [Time frame: 10 years after HSCT]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Other

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT06351462 · APHP200008

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗