Patients with Sickle cell disease
Conditions
Interventions
Twenty patients will recelve weekly intramuscular injections of 10 mg methotrexate for 12 weeks. A supplement of 5 mg folinic acid will be offered per os or intramuscularly 48-72h after each methotrex
Drug
Sponsors
Centro Infantil Boldrini
Centro Infantil Boldrini
Eligibility
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: Patients with sickle cell disease who are under chronic hydroxyurea treatment, have more than 3 vaso-occlusive crises per year, and who experience a current vaso-occlusive crisis that is refractory to opioids for a period longer than 3 weeks. Patients should be older than 18 years. Patients must have signed the Informed Consent Form.
Exclusion criteria
Exclusion criteria: Pregnancy. Ongoing infection at recruitment into the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary outcome is the control of pain associated to vaso-occlusive crises, evaluated by the number of pain episodes and pain intensity during the 12 weeks of methotrexate treatment, compared to an identical pre-treatment period.;Primary Outcome at conclusion of the study: Fourteen patients were recruited into the study. The median McGill Pain Index at week 12 decreased in eight patients (27.2%), was nearly unchanged in two individuals (<3%), and increased in four cases (22.5%) as compared to the values recorded at week 0. Half of the patients (n = 7) had avascular osteonecrosis, 5 of whom had a reduction greater than or equal to 50% of the chronic pain associated to this complication. Altogether, 10 out 14 patients had some degree of response to methotrexate related to pain. Besides the 8 patients that had a reduction of the McGill pain index, 2 individuals experienced a reduction greater than or equal to 50% of the osteonecrosis-associated chronic pain despite lack of improvement in acute pain crises. The median number of acute vaso-occlusive crises that needed medical assistance has not changed significantly during the 12-week treatment as compared to the frequency recorded in an identical pre-treatment period (12 vs 10.5 crises, respectively). | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary outcomes are the blood level of inflammatory markers and quality of life, that will be evaluated at weeks 0, 6, and 12 of the study.;The secondary outcomes at the end of the study were: (i) Inflammatory markers: the blood concentrations of only 2 chemokines out of 16 molecular markers have changed significantly at week 12 as compared to the values recorded at week 0, with a median increase of 56.6% of CXCL10 (P = 0.0463) and 47.5% of CXCL12 (P < 0.0001) and (ii) Quality of life: there was 44.4% median improvement in the SF-36 physical functioning domain subscale reported by the patients at week 12 as compared to week 0 of the study (P = 0.0198). This gain was of 96.9% in the 7 patients with avascular osteonecrosis (P = 0.0048). There was no significant change pattern related to depression in the studied patients as assessed by the Beck Inventory. | — |
Countries
Brazil
Contacts
Public ContactSilvia;Silvia Brandalise;Brandalise
Centro Infantil Boldrini;Centro Infantil Boldrini
Outcome results
None listed